Trial Outcomes & Findings for Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency (NCT NCT01757184)

NCT ID: NCT01757184

Last Updated: 2020-12-29

Results Overview

Alanine aminotransferase (ALT) normalization was defined as an abnormal baseline value (ALT \> the age- and gender-specific upper limit of normal \[ULN\] provided by the central laboratory performing the assay) that becomes normal (\< ULN). Alanine aminotransferase normalization was evaluated at the end of the Double-blind Period (the last double-blind assessment) and at the end of the Open-label Period (last open-label assessment). Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 66 participants
• Primary outcome timeframe: Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256)
• Results posted on: 2020-12-29

Participant Flow

A total of 56 study centers located in 17 countries were initiated in this study. Participants were enrolled and treated at 41 centers in 15 countries, including 35 primary centers where participants initiated treatment and 6 qualified local medical centers where participants who were medically stable were transferred for long-term treatment.

To assess eligibility, participants were screened for a period of up to 6 weeks prior to enrollment in the study. A total of 86 participants were screened. Six of these participants underwent rescreening (of which 2 were eligible for the study). In total, 66 participants were eligible for the study and 20 participants were screen failures.

Participant milestones

Measure	Double-blind Sebelipase Alfa Double-blind Period: Intravenous (IV) infusions of sebelipase alfa at a dose of 1 milligram/kilogram (mg/kg) administered every other week (qow).	Double-blind Placebo Double-blind Period: IV infusions of placebo administered qow.	Open-label Sebelipase Alfa/Sebelipase Alfa Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-label Placebo/Sebelipase Alfa Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Double-blind Period STARTED	36	30	0	0
Double-blind Period Received at Least 1 Dose of Study Drug	36	30	0	0
Double-blind Period COMPLETED	35	30	0	0
Double-blind Period NOT COMPLETED	1	0	0	0
Open-label Period STARTED	0	0	36	30
Open-label Period Received at Least 1 Dose of Study Drug	0	0	36	30
Open-label Period COMPLETED	0	0	32	27
Open-label Period NOT COMPLETED	0	0	4	3

Reasons for withdrawal

Measure	Double-blind Sebelipase Alfa Double-blind Period: Intravenous (IV) infusions of sebelipase alfa at a dose of 1 milligram/kilogram (mg/kg) administered every other week (qow).	Double-blind Placebo Double-blind Period: IV infusions of placebo administered qow.	Open-label Sebelipase Alfa/Sebelipase Alfa Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-label Placebo/Sebelipase Alfa Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Double-blind Period Adverse Event	1	0	0	0
Open-label Period Lost to Follow-up	0	0	2	0
Open-label Period Withdrawal by Subject	0	0	1	1
Open-label Period Discontinued by Sponsor	0	0	1	2

Baseline Characteristics

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

Baseline characteristics by cohort

Measure	Double-Blind Sebelipase Alfa n=36 Participants Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow.	Double-Blind Placebo n=30 Participants Double-blind Period: IV infusions of placebo administered qow.	Total n=66 Participants Total of all reporting groups
Age, Continuous	17.39 years STANDARD_DEVIATION 11.529 • n=5 Participants	15.70 years STANDARD_DEVIATION 10.283 • n=7 Participants	16.62 years STANDARD_DEVIATION 10.930 • n=5 Participants
Sex: Female, Male Female	18 Participants n=5 Participants	15 Participants n=7 Participants	33 Participants n=5 Participants
Sex: Female, Male Male	18 Participants n=5 Participants	15 Participants n=7 Participants	33 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=5 Participants	4 Participants n=7 Participants	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	30 Participants n=5 Participants	26 Participants n=7 Participants	56 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	27 Participants n=5 Participants	28 Participants n=7 Participants	55 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	5 Participants n=5 Participants	2 Participants n=7 Participants	7 Participants n=5 Participants

PRIMARY outcome

Timeframe: Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256)

Population: Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo). Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa).

Alanine aminotransferase (ALT) normalization was defined as an abnormal baseline value (ALT > the age- and gender-specific upper limit of normal [ULN] provided by the central laboratory performing the assay) that becomes normal (< ULN). Alanine aminotransferase normalization was evaluated at the end of the Double-blind Period (the last double-blind assessment) and at the end of the Open-label Period (last open-label assessment). Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings.

Outcome measures

Measure	Double-Blind Sebelipase Alfa n=36 Participants Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow.	Double-Blind Placebo n=30 Participants Double-blind Period: IV infusions of placebo administered qow.	Open-Label Sebelipase Alfa/Sebelipase Alfa n=36 Participants Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-Label Placebo/Sebelipase Alfa n=30 Participants Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Percentage Of Participants Achieving Alanine Aminotransferase Normalization	31 percentage of participants	7 percentage of participants	56 percentage of participants	37 percentage of participants

SECONDARY outcome

Timeframe: Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256).

Population: Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo). Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa).

Relative reduction (percentage change from baseline) in LDL-C, as assessed by laboratory measurements was evaluated at the end of the Double-blind Period and at the end of the Open-label Period. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings.

Outcome measures

Measure	Double-Blind Sebelipase Alfa n=36 Participants Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow.	Double-Blind Placebo n=30 Participants Double-blind Period: IV infusions of placebo administered qow.	Open-Label Sebelipase Alfa/Sebelipase Alfa n=36 Participants Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-Label Placebo/Sebelipase Alfa n=30 Participants Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Percent Change From Baseline In Low-density Lipoprotein Cholesterol (LDL-C)	-28.42 percent change Standard Deviation 22.304	-6.25 percent change Standard Deviation 13.015	-19.74 percent change Standard Deviation 33.262	-18.09 percent change Standard Deviation 33.685

SECONDARY outcome

Timeframe: Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256).

Population: Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo). Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa).

Relative reduction (percent change from baseline) in non-HDL-C, as assessed by laboratory measurements, was evaluated at the end of the Double-blind Period and the Open-label Period. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings.

Outcome measures

Measure	Double-Blind Sebelipase Alfa n=36 Participants Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow.	Double-Blind Placebo n=30 Participants Double-blind Period: IV infusions of placebo administered qow.	Open-Label Sebelipase Alfa/Sebelipase Alfa n=36 Participants Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-Label Placebo/Sebelipase Alfa n=30 Participants Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Percent Change From Baseline In Non-high Density Lipoprotein Cholesterol (Non-HDL-C)	-27.97 percent change Standard Deviation 18.612	-6.94 percent change Standard Deviation 10.922	-19.75 percent change Standard Deviation 26.875	-18.34 percent change Standard Deviation 29.177

SECONDARY outcome

Timeframe: Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256).

Population: Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo). Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa).

Aspartate aminotransferase (AST) normalization was defined as an abnormal baseline value (AST > the age- and gender-specific ULN provided by the central laboratory performing the assay) that becomes normal (< ULN). AST normalization was evaluated at the end of the Double-blind Period (the last Double-blind assessment) and at the end of the Open-label Period (last open-label assessment).

Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings.

Outcome measures

Measure	Double-Blind Sebelipase Alfa n=36 Participants Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow.	Double-Blind Placebo n=29 Participants Double-blind Period: IV infusions of placebo administered qow.	Open-Label Sebelipase Alfa/Sebelipase Alfa n=36 Participants Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-Label Placebo/Sebelipase Alfa n=29 Participants Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Percentage Of Participants Achieving Aspartate Aminotransferase Normalization	42 percentage of participants	3 percentage of participants	69 percentage of participants	62 percentage of participants

SECONDARY outcome

Timeframe: Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256).

Population: Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo). Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa).

Relative reduction (percent change from baseline) in triglycerides, as assessed by laboratory measurements, was evaluated at the end of the Double-blind Period and the Open-label Period. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings.

Outcome measures

Measure	Double-Blind Sebelipase Alfa n=36 Participants Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow.	Double-Blind Placebo n=30 Participants Double-blind Period: IV infusions of placebo administered qow.	Open-Label Sebelipase Alfa/Sebelipase Alfa n=36 Participants Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-Label Placebo/Sebelipase Alfa n=30 Participants Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Percent Change From Baseline In Triglycerides	-25.45 percent change Standard Deviation 29.411	-11.14 percent change Standard Deviation 28.827	-11.87 percent change Standard Deviation 34.580	-19.63 percent change Standard Deviation 27.066

SECONDARY outcome

Timeframe: Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256).

Population: Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo). Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa).

Relative increase (percent change from baseline) in HDL-C, assessed by laboratory measurements, was evaluated at the end of the Double-blind Period and the Open-label Period. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings.

Outcome measures

Measure	Double-Blind Sebelipase Alfa n=36 Participants Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow.	Double-Blind Placebo n=30 Participants Double-blind Period: IV infusions of placebo administered qow.	Open-Label Sebelipase Alfa/Sebelipase Alfa n=36 Participants Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-Label Placebo/Sebelipase Alfa n=30 Participants Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Percent Change From Baseline In High-density Lipoprotein Cholesterol (HDL-C)	19.57 percent change Standard Deviation 16.833	-0.29 percent change Standard Deviation 12.360	31.65 percent change Standard Deviation 28.971	34.78 percent change Standard Deviation 29.927

SECONDARY outcome

Timeframe: Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256).

Population: Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo). Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa).

Decrease in liver fat content, as assessed by magnetic resonance imaging (MRI), was evaluated in participants for whom imaging was performed. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings.

Outcome measures

Measure	Double-Blind Sebelipase Alfa n=32 Participants Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow.	Double-Blind Placebo n=25 Participants Double-blind Period: IV infusions of placebo administered qow.	Open-Label Sebelipase Alfa/Sebelipase Alfa n=35 Participants Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-Label Placebo/Sebelipase Alfa n=25 Participants Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Percent Change From Baseline In Liver Fat Content	-31.98 percent change Standard Deviation 26.763	-4.21 percent change Standard Deviation 15.559	-9.89 percent change Standard Deviation 32.892	-0.93 percent change Standard Deviation 37.233

SECONDARY outcome

Timeframe: Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline up to Week 52.

Population: Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo). Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa).

The number of participants who had an improvement in hepatic histopathology (defined as a decrease of > 5% in hepatic steatosis score, assessed by morphometry), as determined by blinded central pathologist review, in the participants for whom liver biopsy was performed. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group.

Outcome measures

Measure	Double-Blind Sebelipase Alfa n=16 Participants Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow.	Double-Blind Placebo n=10 Participants Double-blind Period: IV infusions of placebo administered qow.	Open-Label Sebelipase Alfa/Sebelipase Alfa n=12 Participants Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-Label Placebo/Sebelipase Alfa n=6 Participants Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Participants With Improvement In Liver Histopathology (Decrease Of > 5% In Hepatic Steatosis Score)	10 participants	4 participants	7 participants	4 participants

SECONDARY outcome

Timeframe: Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256).

Population: Double-blind Period: All participants in the Full Analysis Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa or placebo). Open-Label Period: All participants in the Extension Set (defined as participants who received at least 1 dose \[or any portion of a dose\] of sebelipase alfa).

Relative reduction (percent change from baseline) in liver volume, as assessed by MRI, was evaluated in participants for whom imaging was performed. Baseline for the Open-label Period was defined relative to the first infusion of sebelipase alfa, which occurred at Week 0 for participants in the sebelipase alfa/sebelipase alfa group and Week 22 for participants in the placebo/sebelipase alfa group. The last open-label assessment varied by participant, depending on whether a participant completed treatment through Week 256 or discontinued prior to this timepoint to transition out of clinical study settings.

Outcome measures

Measure	Double-Blind Sebelipase Alfa n=33 Participants Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow.	Double-Blind Placebo n=27 Participants Double-blind Period: IV infusions of placebo administered qow.	Open-Label Sebelipase Alfa/Sebelipase Alfa n=36 Participants Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-Label Placebo/Sebelipase Alfa n=27 Participants Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Percent Change From Baseline In Liver Volume	-10.28 percent change Standard Deviation 10.510	-2.66 percent change Standard Deviation 10.107	-24.04 percent change Standard Deviation 15.792	-21.55 percent change Standard Deviation 11.727

Adverse Events

Double-blind Sebelipase Alfa

Serious events: 2 serious events

Other events: 31 other events

Deaths: 0 deaths

Double-blind Placebo

Serious events: 1 serious events

Other events: 28 other events

Deaths: 0 deaths

Open-label Sebelipase Alfa/Sebelipase Alfa

Serious events: 6 serious events

Other events: 35 other events

Deaths: 0 deaths

Open-label Placebo/Sebelipase Alfa

Serious events: 5 serious events

Other events: 29 other events

Deaths: 0 deaths

Serious adverse events

Measure	Double-blind Sebelipase Alfa n=36 participants at risk Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow.	Double-blind Placebo n=30 participants at risk Double-blind Period: IV infusions of placebo administered qow.	Open-label Sebelipase Alfa/Sebelipase Alfa n=36 participants at risk Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-label Placebo/Sebelipase Alfa n=30 participants at risk Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Eye disorders Eyelid oedema	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Gastritis	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Gastrointestinal haemorrhage	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
General disorders Chest discomfort	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Appendicitis	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Gastroenteritis	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Influenza	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Peritonitis	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Infusion-related reaction	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Post procedural haemorrhage	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Procedural pain	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Road traffic accident	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Musculoskeletal and connective tissue disorders Patellofemoral pain syndrome	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Musculoskeletal and connective tissue disorders Plantar fasciitis	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Hepatic cancer	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Metastases to lung	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Nervous system disorders Epilepsy	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Nervous system disorders Hepatic encephalopathy	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Respiratory, thoracic and mediastinal disorders Sleep apnoea syndrome	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Skin and subcutaneous tissue disorders Urticaria	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Vascular disorders Hyperaemia	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.

Other adverse events

Measure	Double-blind Sebelipase Alfa n=36 participants at risk Double-blind Period: IV infusions of sebelipase alfa at a dose of 1 mg/kg administered qow.	Double-blind Placebo n=30 participants at risk Double-blind Period: IV infusions of placebo administered qow.	Open-label Sebelipase Alfa/Sebelipase Alfa n=36 participants at risk Participants who were randomized to receive sebelipase alfa during the Double-blind Period and also received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.	Open-label Placebo/Sebelipase Alfa n=30 participants at risk Participants who were randomized to receive placebo during the Double-blind Period and received sebelipase alfa in the Open-label Period. All participants received sebelipase alfa at a dose of 1 mg/kg qow, irrespective of treatment received in the Double-blind Period.
Skin and subcutaneous tissue disorders Pruritus	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Diarrhoea	16.7% 6/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	16.7% 5/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	19.4% 7/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	36.7% 11/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Abdominal pain	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	19.4% 7/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	30.0% 9/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Constipation	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	11.1% 4/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.3% 4/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Nausea	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.9% 5/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Vomiting	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	25.0% 9/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	23.3% 7/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Abdominal pain upper	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	27.8% 10/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Upper respiratory tract infection	16.7% 6/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	20.0% 6/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	27.8% 10/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	16.7% 5/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Nasopharyngitis	11.1% 4/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	47.2% 17/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	46.7% 14/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Rhinitis	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	11.1% 4/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	30.0% 9/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Sinusitis	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	11.1% 4/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Tonsillitis	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.3% 4/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	11.1% 4/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Pharyngitis	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	16.7% 5/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	16.7% 6/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	16.7% 5/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Respiratory tract infection viral	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.3% 4/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Varicella	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	16.7% 6/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.9% 5/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	20.0% 6/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Respiratory, thoracic and mediastinal disorders Epistaxis	11.1% 4/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	20.0% 6/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.9% 5/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	16.7% 5/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Respiratory, thoracic and mediastinal disorders Cough	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	33.3% 12/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	30.0% 9/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	22.2% 8/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	16.7% 5/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
General disorders Pyrexia	19.4% 7/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	20.0% 6/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	41.7% 15/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	30.0% 9/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
General disorders Asthenia	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
General disorders Fatigue	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
General disorders Vaccination site pain	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Nervous system disorders Headache	27.8% 10/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	20.0% 6/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	52.8% 19/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	40.0% 12/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Nervous system disorders Syncope	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Procedural Pain	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Skin and subcutaneous tissue disorders Rash	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.3% 4/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Investigations Body temperature increased	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Musculoskeletal and connective tissue disorders Arthralgia	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.3% 4/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Psychiatric disorders Anxiety	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Cardiac disorders Arrhythmia	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Respiratory tract infection	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	16.7% 5/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Gastroenteritis	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.9% 5/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	23.3% 7/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Bronchitis	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Influenza	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Urinary tract infection	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Conjunctivitis	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Oral herpes	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Vulvovaginitis	0.00% 0/18 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/15 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 1/18 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.3% 2/15 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Otitis media	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Viral infection	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Gastroenteritis viral	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Gastrointestinal infection	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Ear infection	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Infections and infestations Rotavirus infection	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Odynophagia	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Toothache	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Abdominal discomfort	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Gastrooesophageal reflux disease	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Eructation	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Gingival bleeding	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Respiratory, thoracic and mediastinal disorders Asthma	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	11.1% 4/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	16.7% 5/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Respiratory, thoracic and mediastinal disorders Nasal congestion	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.9% 5/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Respiratory, thoracic and mediastinal disorders Productive cough	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Nervous system disorders Dizziness	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	16.7% 6/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Nervous system disorders Somnolence	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Skin and subcutaneous tissue disorders Urticaria	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.3% 4/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Skin and subcutaneous tissue disorders Dermatitis allergic	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	20.0% 6/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Skin and subcutaneous tissue disorders Ecchymosis	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Skin and subcutaneous tissue disorders Dermatitis atopic	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Skin and subcutaneous tissue disorders Eczema	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Skin and subcutaneous tissue disorders Dermatitis	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
General disorders Chest pain	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.3% 4/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
General disorders Malaise	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Ligament sprain	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	11.1% 4/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Contusion	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.9% 5/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Skin abrasion	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	11.1% 4/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Arthropod bite	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Laceration	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Thermal burn	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Joint injury	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Injury, poisoning and procedural complications Sunburn	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Musculoskeletal and connective tissue disorders Tendonitis	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Musculoskeletal and connective tissue disorders Pain in extremity	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Musculoskeletal and connective tissue disorders Myalgia	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	10.0% 3/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Musculoskeletal and connective tissue disorders Neck pain	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Musculoskeletal and connective tissue disorders Osteoporosis	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Reproductive system and breast disorders Dysmenorrhoea	0.00% 0/18 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 1/15 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	16.7% 3/18 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	26.7% 4/15 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Investigations Blood cholesterol increased	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Investigations Blood pressure increased	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Investigations Cardiac murmur	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Investigations Eosinophil count increased	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Metabolism and nutrition disorders Vitamin D deficiency	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	22.2% 8/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	13.3% 4/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Metabolism and nutrition disorders Iron deficiency	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	8.3% 3/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Blood and lymphatic system disorders Anaemia	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	3.3% 1/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Blood and lymphatic system disorders Eosinophilia	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Immune system disorders Seasonal allergy	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	11.1% 4/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Psychiatric disorders Autism spectrum disorder	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Psychiatric disorders Sleep disorder	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Ear and labyrinth disorders Ear pain	0.00% 0/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	6.7% 2/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Vascular disorders Hypertension	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.
Gastrointestinal disorders Gastritis	2.8% 1/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	5.6% 2/36 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.	0.00% 0/30 • In the Double-blind Period, adverse events were assessed on or after the first infusion of study drug until Week 20. In the Open-label Period, adverse events were assessed after the first infusion of study drug on Week 22 up to Follow-up call (4 weeks [+ 7 days] after the last infusion of sebelipase alfa). Adverse events were obtained through spontaneous reporting or were elicited by specific questioning of the participant or the participant's parent or legal guardian.

Additional Information

Alexion Pharmaceuticals, Inc.

Alexion Pharmaceuticals, Inc.

Phone: 855-752-2356

Email: clinicaltrials@alexion.com

Results disclosure agreements

• Principal investigator is a sponsor employee Institution and/or Principal Investigator may publish or make an individual oral or written presentation of publication of study results either 1) after Alexion publishes the study data or 2) 18 months after the completion of the study, whichever comes first. Alexion reserves the right to review and comment on a communication at least 30 days prior to submission with a provision to extend the review for an additional 60 days where information is relevant to a patent application.
• Publication restrictions are in place

Restriction type: OTHER