Trial Outcomes & Findings for Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age (NCT NCT02418455)

Last Updated: 2019-10-30

Results Overview

Liquid chromatography-mass spectrometry/mass spectrometry-dermatan sulfate (LS-MS/MS-DS) method. For the participant previously treated with UX003 under an eIND, percent change from initial baseline was used.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

Baseline (Week 0), Week 48

Results posted on

2019-10-30

Participant Flow

Participant milestones

Participant milestones
Measure	UX003 UX003 4 mg/kg every other week (QOW). Initial treatment period 48 weeks. Continuation period up to 240 weeks.
48-Week Treatment Period STARTED	8
48-Week Treatment Period Emergency IND (eIND)	1
48-Week Treatment Period COMPLETED	7
48-Week Treatment Period NOT COMPLETED	1
Continuation Period STARTED	7
Continuation Period COMPLETED	0
Continuation Period NOT COMPLETED	7

Reasons for withdrawal

Reasons for withdrawal
Measure	UX003 UX003 4 mg/kg every other week (QOW). Initial treatment period 48 weeks. Continuation period up to 240 weeks.
48-Week Treatment Period Other- Not Specified	1
Continuation Period Sponsor Decision	7

Baseline Characteristics

eIND and non-eIND participants were analyzed separately for this measure.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	UX003 n=8 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Age, Continuous	3.25 years STANDARD_DEVIATION 1.197 • n=8 Participants
Sex: Female, Male Female	3 Participants n=8 Participants
Sex: Female, Male Male	5 Participants n=8 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=8 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=8 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=8 Participants
Race/Ethnicity, Customized Asian	2 Participants n=8 Participants
Race/Ethnicity, Customized Black or African American	1 Participants n=8 Participants
Race/Ethnicity, Customized White	3 Participants n=8 Participants
Race/Ethnicity, Customized Other, Not Specified	2 Participants n=8 Participants
Urinary Glycosaminoglycans (uGAG) Excretion	2.092 g GAG/g creatinine STANDARD_DEVIATION 1.3307 • n=8 Participants
Standing Height eIND	52.20 cm STANDARD_DEVIATION NA • n=1 Participants • eIND and non-eIND participants were analyzed separately for this measure.
Standing Height non-eIND	89.34 cm STANDARD_DEVIATION 7.198 • n=7 Participants • eIND and non-eIND participants were analyzed separately for this measure.
Standing Height Z-Score eIND	-5.352 Z-score STANDARD_DEVIATION NA • n=1 Participants • eIND and non-eIND participants were analyzed separately for this measure.
Standing Height Z-Score Non-eIND	-2.241 Z-score STANDARD_DEVIATION 0.4327 • n=7 Participants • eIND and non-eIND participants were analyzed separately for this measure.
Head Circumference eIND	37.00 cm STANDARD_DEVIATION NA • n=1 Participants • eIND and non-eIND participants were analyzed separately for this measure.
Head Circumference Non-eIND	51.57 cm STANDARD_DEVIATION 2.130 • n=7 Participants • eIND and non-eIND participants were analyzed separately for this measure.
Head Circumference Z-Score eIND	-3.329 Z-score STANDARD_DEVIATION NA • n=1 Participants • eIND and non-eIND participants were analyzed separately for this measure. Participants with a non-missing baseline assessment are presented.
Head Circumference Z-Score Non-eIND	1.465 Z-score STANDARD_DEVIATION 1.3186 • n=5 Participants • eIND and non-eIND participants were analyzed separately for this measure. Participants with a non-missing baseline assessment are presented.
Weight eIND	4.26 kg STANDARD_DEVIATION NA • n=1 Participants • eIND and non-eIND participants were analyzed separately for this measure.
Weight Non-eIND	13.99 kg STANDARD_DEVIATION 2.274 • n=7 Participants • eIND and non-eIND participants were analyzed separately for this measure.
Historical Pre-Treatment (Within 2 Years) Growth Velocity	5.06 cm/year STANDARD_DEVIATION 1.885 • n=5 Participants • All enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline.
Pre-Treatment (Within 2 Years) Growth Velocity Z-Score	-2.587 Z-score STANDARD_DEVIATION 1.4860 • n=4 Participants • All enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline. Growth velocity Z-score was only calculated for participants ≥ 2.25 years.
Liver Measurement eIND	7.60 cm STANDARD_DEVIATION NA • n=1 Participants • eIND and non-eIND participants were analyzed separately for this measure. One participant in the non-eIND group did not have a baseline measurement.
Liver Measurement Non-eIND	10.70 cm STANDARD_DEVIATION 1.138 • n=6 Participants • eIND and non-eIND participants were analyzed separately for this measure. One participant in the non-eIND group did not have a baseline measurement.
Spleen Measurement eIND	6.40 cm STANDARD_DEVIATION NA • n=1 Participants • e-IND and non-eIND participants were analyzed separately
Spleen Measurement Non-eIND	8.17 cm STANDARD_DEVIATION 1.115 • n=7 Participants • e-IND and non-eIND participants were analyzed separately

PRIMARY outcome

Timeframe: Baseline (Week 0), Week 48

Population: Full analysis set: all enrolled participants who received at least one dose of UX003 during the study and had a non-missing baseline and Week 48 assessment.

Outcome measures

Outcome measures
Measure	UX003 n=7 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.	UX003: Non-eIND UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Percent Change From Baseline in uGAG Excretion (LC-MS/MS-DS) at Week 48	-58.17 percent change Standard Deviation 16.915	—

PRIMARY outcome

Timeframe: From first dose of study drug until 30 days after the last dose of study drug. Mean (SD) treatment duration was 98.11 (29.02) weeks

Population: Full analysis set: all enrolled participants who received at least one dose of UX003 during the study.

Adverse event (AE): any untoward medical occurrence in a participant, whether or not considered drug related. Serious AE (SAE): an AE or suspected adverse reaction that at any dose results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; a congenital anomaly/birth defect. Other important medical events may also, in the opinion of the Investigator, be considered SAEs. An AE was considered a TEAE if it occurred on or after the first dose, and was not present prior to the first dose, or it was present at the first dose but increased in severity during the study. Events recorded as either possibly, probably, or definitely related to treatment were categorized as related. AE severity was graded using the National Cancer Institute's Common Terminology Criteria for Adverse Events, Version 4.03.

Outcome measures

Outcome measures
Measure	UX003 n=8 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.	UX003: Non-eIND UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs TEAEs	8 Participants	—
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs Serious TEAEs	3 Participants	—
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs Treatment-Related TEAEs	5 Participants	—
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs Treatment-Related Serious TEAEs	1 Participants	—
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs Grade 3 or 4 TEAEs	2 Participants	—
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs TEAEs Leading to Treatment Discontinuation	0 Participants	—
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs TEAEs Leading to Study Discontinuation	0 Participants	—
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and Discontinuations Due to TEAEs TEAEs Leading to Death	0 Participants	—

SECONDARY outcome

Timeframe: Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132

Population: Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point.

For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline.

Outcome measures

Outcome measures
Measure	UX003 n=1 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.	UX003: Non-eIND n=7 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Change From Baseline Over Time in Standing Height Change at Week 36	20.00 cm Standard Deviation NA 1 participant analyzed	5.64 cm Standard Deviation 1.725
Change From Baseline Over Time in Standing Height Change at Week 12	—	1.94 cm Standard Deviation 1.927
Change From Baseline Over Time in Standing Height Change at Week 24	15.00 cm Standard Deviation NA 1 participant analyzed	3.71 cm Standard Deviation 0.994
Change From Baseline Over Time in Standing Height Change at Week 48	—	6.57 cm Standard Deviation 1.058
Change From Baseline Over Time in Standing Height Change at Week 60	—	8.59 cm Standard Deviation 1.318
Change From Baseline Over Time in Standing Height Change at Week 72	—	9.63 cm Standard Deviation 1.451
Change From Baseline Over Time in Standing Height Change at Week 84	—	10.93 cm Standard Deviation 2.095
Change From Baseline Over Time in Standing Height Change at Week 96	—	11.86 cm Standard Deviation 3.146
Change From Baseline Over Time in Standing Height Change at Week 108	—	12.17 cm Standard Deviation 3.329
Change From Baseline Over Time in Standing Height Change at Week 120	—	12.67 cm Standard Deviation 3.617
Change From Baseline Over Time in Standing Height Change at Week 132	—	14.80 cm Standard Deviation NA 1 participant analyzed

SECONDARY outcome

Timeframe: Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132

Population: Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point.

The Z-score indicates the number of standard deviations away from a reference population (from the CDC growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline.

Outcome measures

Outcome measures
Measure	UX003 n=1 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.	UX003: Non-eIND n=7 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Change From Baseline Over Time in Standing Height Z-Score Change at Week 12	—	0.106 Z-score Standard Deviation 0.4938
Change From Baseline Over Time in Standing Height Z-Score Change at Week 24	-0.481 Z-score Standard Deviation NA 1 participant analyzed	0.188 Z-score Standard Deviation 0.3013
Change From Baseline Over Time in Standing Height Z-Score Change at Week 36	0.314 Z-score Standard Deviation NA 1 participant analyzed	0.314 Z-score Standard Deviation 0.4103
Change From Baseline Over Time in Standing Height Z-Score Change at Week 48	—	0.196 Z-score Standard Deviation 0.3012
Change From Baseline Over Time in Standing Height Z-Score Change at Week 60	—	0.333 Z-score Standard Deviation 0.3883
Change From Baseline Over Time in Standing Height Z-Score Change at Week 72	—	0.246 Z-score Standard Deviation 0.3299
Change From Baseline Over Time in Standing Height Z-Score Change at Week 84	—	0.203 Z-score Standard Deviation 0.3973
Change From Baseline Over Time in Standing Height Z-Score Change at Week 96	—	0.237 Z-score Standard Deviation 0.5312
Change From Baseline Over Time in Standing Height Z-Score Change at Week 108	—	-0.035 Z-score Standard Deviation 0.4531
Change From Baseline Over Time in Standing Height Z-Score Change at Week 120	—	-0.176 Z-score Standard Deviation 0.4915
Change From Baseline Over Time in Standing Height Z-Score Change at Week 132	—	-0.147 Z-score Standard Deviation NA 1 participant analyzed

SECONDARY outcome

Timeframe: Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132

Population: Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point.

For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline.

Outcome measures

Outcome measures
Measure	UX003 n=1 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.	UX003: Non-eIND n=7 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Change From Baseline Over Time in Head Circumference Change at Week 12	—	0.36 cm Standard Deviation 0.378
Change From Baseline Over Time in Head Circumference Change at Week 24	12.50 cm Standard Deviation NA 1 participant analyzed	0.54 cm Standard Deviation 0.360
Change From Baseline Over Time in Head Circumference Change at Week 36	12.00 cm Standard Deviation NA 1 participant analyzed	-0.27 cm Standard Deviation 1.732
Change From Baseline Over Time in Head Circumference Change at Week 48	—	0.79 cm Standard Deviation 0.267
Change From Baseline Over Time in Head Circumference Change at Week 60	—	0.86 cm Standard Deviation 0.378
Change From Baseline Over Time in Head Circumference Change at Week 72	—	1.10 cm Standard Deviation 0.224
Change From Baseline Over Time in Head Circumference Change at Week 84	—	0.70 cm Standard Deviation 0.837
Change From Baseline Over Time in Head Circumference Change at Week 96	—	0.84 cm Standard Deviation 1.108
Change From Baseline Over Time in Head Circumference Change at Week 108	—	1.07 cm Standard Deviation 1.793
Change From Baseline Over Time in Head Circumference Change at Week 120	—	1.33 cm Standard Deviation 2.082
Change From Baseline Over Time in Head Circumference Change at Week 132	—	2.00 cm Standard Deviation NA 1 participant analyzed

SECONDARY outcome

Timeframe: Baseline, Weeks 12, 24, 36, 48

Population: Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point.

Outcome measures

Outcome measures
Measure	UX003 n=1 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.	UX003: Non-eIND n=1 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Change From Baseline Over Time in Head Circumference Z-Score Change at Week 12	—	0.052 Z-score Standard Deviation NA 1 participant analyzed
Change From Baseline Over Time in Head Circumference Z-Score Change at Week 24	3.804 Z-score Standard Deviation NA 1 participant analyzed	0.177 Z-score Standard Deviation NA 1 participant analyzed
Change From Baseline Over Time in Head Circumference Z-Score Change at Week 36	3.263 Z-score Standard Deviation NA 1 participant analyzed	-0.733 Z-score Standard Deviation NA 1 participant analyzed
Change From Baseline Over Time in Head Circumference Z-Score Change at Week 48	—	-0.218 Z-score Standard Deviation NA 1 participant analyzed

SECONDARY outcome

Timeframe: Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132

Population: Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point.

For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline.

Outcome measures

Outcome measures
Measure	UX003 n=1 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.	UX003: Non-eIND n=7 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Change From Baseline Over Time in Weight Change at Week 24	5.94 kg Standard Deviation NA 1 participant analyzed	1.21 kg Standard Deviation 0.904
Change From Baseline Over Time in Weight Change at Week 12	5.54 kg Standard Deviation NA 1 participant analyzed	0.69 kg Standard Deviation 0.626
Change From Baseline Over Time in Weight Change at Week 36	5.94 kg Standard Deviation NA 1 participant analyzed	1.46 kg Standard Deviation 1.416
Change From Baseline Over Time in Weight Change at Week 48	6.34 kg Standard Deviation NA 1 participant analyzed	2.11 kg Standard Deviation 1.110
Change From Baseline Over Time in Weight Change at Week 60	—	2.91 kg Standard Deviation 1.447
Change From Baseline Over Time in Weight Change at Week 72	—	3.49 kg Standard Deviation 2.084
Change From Baseline Over Time in Weight Change at Week 84	—	3.63 kg Standard Deviation 2.170
Change From Baseline Over Time in Weight Change at Week 96	—	5.06 kg Standard Deviation 2.889
Change From Baseline Over Time in Weight Change at Week 108	—	4.53 kg Standard Deviation 3.415
Change From Baseline Over Time in Weight Change at Week 120	—	5.17 kg Standard Deviation 3.968
Change From Baseline Over Time in Weight Change at Week 132	—	5.70 kg Standard Deviation NA 1 participant analyzed

SECONDARY outcome

Timeframe: Pre-treatment (based on standing height within 2 years prior to treatment), Post-treatment (based on all standing height data during the study period up to 240 weeks)

Population: Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with both historical pre-treatment (within 2 years) and post-treatment data.

The growth velocity for pre-treatment is based on standing height within 2 years prior to treatment. The growth velocity for post-treatment is based on all standing height data during the study period. For the participant previously treated with UX003 under an eIND, the growth velocity was calculated for pre initial UX003 treatment and post initial UX003 treatment.

Outcome measures

Outcome measures
Measure	UX003 n=5 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.	UX003: Non-eIND UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Post-UX003 Growth Velocity (cm/yr) for Participants With Both Historical Pre-UX003 (Within 2 Years) and Post-UX003 Data	6.20 cm/year Standard Deviation 1.954	—

SECONDARY outcome

Timeframe: Pre-treatment (based on standing height within 2 years prior to treatment), Post-treatment (based on all standing height data during the study period up to Week 48)

Population: Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with both historical pre-treatment (within 2 years) and post-treatment data. Growth velocity Z-score was only calculated for participants ≥ 2.25 years.

The Z-score indicates the number of standard deviations away from a reference population (based on Tanner's standard \[Tanner et al. 1985\]) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. The growth velocity for pre-treatment is based on standing height within 2 years prior to treatment. The growth velocity for post-treatment is based on all standing height data during the study period. For the participant previously treated with UX003 under an eIND, the growth velocity was calculated for pre initial UX003 treatment and post initial UX003 treatment.

Outcome measures

Outcome measures
Measure	UX003 n=4 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.	UX003: Non-eIND UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Change From Pre-Treatment (Within 2 Years) to Post-Treatment Growth Velocity Z-Score	2.291 Z-score Standard Deviation 3.3555	—

SECONDARY outcome

Timeframe: Baseline, Weeks 12, 24, 48, 96, 144

Population: Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point.

For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline.

Outcome measures

Outcome measures
Measure	UX003 n=1 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.	UX003: Non-eIND n=6 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Change From Baseline Over Time in Liver Measurement Change at Week 12	-0.30 cm Standard Deviation NA 1 participant analyzed	-0.87 cm Standard Deviation 1.873
Change From Baseline Over Time in Liver Measurement Change at Week 24	0.70 cm Standard Deviation NA 1 participant analyzed	-1.16 cm Standard Deviation 2.701
Change From Baseline Over Time in Liver Measurement Change at Week 48	-0.40 cm Standard Deviation NA 1 participant analyzed	-0.85 cm Standard Deviation 2.160
Change From Baseline Over Time in Liver Measurement Change at Week 96	—	-0.45 cm Standard Deviation 1.868
Change From Baseline Over Time in Liver Measurement Change at Week 144	—	-1.50 cm Standard Deviation 3.394

SECONDARY outcome

Timeframe: Baseline, Weeks 12, 24, 48, 96, 144

Population: Full analysis set: all enrolled participants who received at least one dose of UX003 during the study with a non-missing assessment at baseline and given time point.

For all participants (including the participant previously treated with UX003 under an eIND), the last non-missing study assessment prior to the first dose in this study was used as baseline.

Outcome measures

Outcome measures
Measure	UX003 n=1 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.	UX003: Non-eIND n=7 Participants UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Change From Baseline Over Time in Spleen Measurement Change at Week 12	0.00 cm Standard Deviation NA 1 participant analyzed	-0.37 cm Standard Deviation 0.624
Change From Baseline Over Time in Spleen Measurement Change at Week 24	-0.10 cm Standard Deviation NA 1 participant analyzed	-0.03 cm Standard Deviation 1.493
Change From Baseline Over Time in Spleen Measurement Change at Week 48	-1.60 cm Standard Deviation NA 1 participant analyzed	-0.16 cm Standard Deviation 1.128
Change From Baseline Over Time in Spleen Measurement Change at Week 96	—	0.21 cm Standard Deviation 1.056
Change From Baseline Over Time in Spleen Measurement Change at Week 144	—	-1.12 cm Standard Deviation 0.686

Adverse Events

UX003

Serious events: 3 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	UX003 n=8 participants at risk UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Cardiac disorders Bradycardia	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
General disorders Device Fastener Issue	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Otitis Media	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Investigations Oxygen Saturation Decreased	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Musculoskeletal and connective tissue disorders Cervical Spinal Stenosis	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Musculoskeletal and connective tissue disorders Spinal Column Stenosis	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Musculoskeletal and connective tissue disorders Spinal Instability	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Nervous system disorders Cervical Cord Compression	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Nervous system disorders Febrile Convulsion	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Adenoidal Hypertrophy	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.

Other adverse events

Other adverse events
Measure	UX003 n=8 participants at risk UX003 4 mg/kg QOW. Initial treatment period 48 weeks. Continuation period up to 240 weeks.
Blood and lymphatic system disorders Anaemia	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Blood and lymphatic system disorders Lymphadenitis	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Blood and lymphatic system disorders Microcytosis	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Blood and lymphatic system disorders Thrombocytopenia	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Cardiac disorders Bradycardia	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Cardiac disorders Pulmonary Valve Stenosis	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Cardiac disorders Right Ventricular Hypertension	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Cardiac disorders Supraventricular Extrasystoles	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Cardiac disorders Tachycardia	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Congenital, familial and genetic disorders Mucopolysaccharidosis Vii	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Congenital, familial and genetic disorders Pectus Carinatum	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Ear and labyrinth disorders Cerumen Impaction	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Ear and labyrinth disorders Ear Pain	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Eye disorders Corneal Deposits	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Eye disorders Myopia	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Eye disorders Strabismus	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Gastrointestinal disorders Abdominal Pain	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Gastrointestinal disorders Abdominal Pain Upper	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Gastrointestinal disorders Constipation	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Gastrointestinal disorders Diarrhoea	50.0% 4/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Gastrointestinal disorders Gingival Bleeding	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Gastrointestinal disorders Gingival Pain	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Gastrointestinal disorders Lip Haemorrhage	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Gastrointestinal disorders Nausea	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Gastrointestinal disorders Oral Papule	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Gastrointestinal disorders Vomiting	50.0% 4/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
General disorders Device Defective	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
General disorders Device Malfunction	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
General disorders Discomfort	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
General disorders Extravasation	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
General disorders Inflammation	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
General disorders Infusion Site Extravasation	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
General disorders Pyrexia	62.5% 5/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
General disorders Secretion Discharge	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
General disorders Tenderness	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Hepatobiliary disorders Hepatomegaly	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Immune system disorders Anaphylactoid Reaction	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Adenoiditis	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Bacterial Disease Carrier	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Device Related Infection	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Ear Infection	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Enterovirus Infection	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Eye Infection	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Fungal Infection	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Fungal Skin Infection	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Gastroenteritis	50.0% 4/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Gastroenteritis Viral	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Gastrointestinal Infection	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Gingival Abscess	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Nasopharyngitis	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Otitis Media	37.5% 3/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Otitis Media Acute	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Otitis Media Chronic	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Pharyngitis	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Pharyngitis Streptococcal	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Pharyngotonsillitis	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Pneumonia Viral	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Respiratory Tract Infection Viral	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Rhinitis	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Rhinovirus Infection	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Sinusitis	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Tracheitis	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Infections and infestations Upper Respiratory Tract Infection	37.5% 3/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Injury, poisoning and procedural complications Contusion	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Injury, poisoning and procedural complications Fall	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Injury, poisoning and procedural complications Gastrostomy Tube Site Complication	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Injury, poisoning and procedural complications Limb Injury	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Injury, poisoning and procedural complications Procedural Pain	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Injury, poisoning and procedural complications Scar	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Injury, poisoning and procedural complications Scratch	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Injury, poisoning and procedural complications Skin Abrasion	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Injury, poisoning and procedural complications Sunburn	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Investigations Alanine Aminotransferase Increased	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Investigations Blood Calcium Decreased	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Investigations Blood Creatinine Decreased	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Investigations Blood Pressure Increased	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Investigations Body Temperature Increased	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Investigations Body Temperature Abnormal	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Investigations Gamma-Glutamyltransferase Increased	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Investigations Hepatic Enzyme Increased	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Investigations Oxygen Saturation Decreased	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Investigations Right Ventricular Systolic Pressure Increased	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Investigations Vitamin D Decreased	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Musculoskeletal and connective tissue disorders Arthralgia	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Musculoskeletal and connective tissue disorders Bone Deformity	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Musculoskeletal and connective tissue disorders Muscle Spasms	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Musculoskeletal and connective tissue disorders Muscular Weakness	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Musculoskeletal and connective tissue disorders Musculoskeletal Chest Pain	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Musculoskeletal and connective tissue disorders Musculoskeletal Pain	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Musculoskeletal and connective tissue disorders Pain In Extremity	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Musculoskeletal and connective tissue disorders Spinal Deformity	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Nervous system disorders Cerebellar Microhaemorrhage	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Nervous system disorders Enlarged Cerebral Perivascular Spaces	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Nervous system disorders Headache	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Nervous system disorders Lethargy	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Nervous system disorders Myelopathy	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Nervous system disorders Periodic Limb Movement Disorder	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Psychiatric disorders Attention Deficit/Hyperactivity Disorder	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Psychiatric disorders Irritability	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Psychiatric disorders Sleep Disorder	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Renal and urinary disorders Bladder Disorder	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Renal and urinary disorders Haematuria	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Asthma	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Cough	37.5% 3/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Hypoxia	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Nasal Congestion	25.0% 2/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Nasal Mucosal Disorder	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Oropharyngeal Pain	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Pneumonia Aspiration	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Rhinitis Allergic	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	37.5% 3/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Tonsillar Hypertrophy	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Upper Respiratory Tract Congestion	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Respiratory, thoracic and mediastinal disorders Wheezing	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Skin and subcutaneous tissue disorders Alopecia	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Skin and subcutaneous tissue disorders Blood Blister	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Skin and subcutaneous tissue disorders Dermatitis	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Skin and subcutaneous tissue disorders Ecchymosis	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Skin and subcutaneous tissue disorders Intertrigo	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Skin and subcutaneous tissue disorders Keloid Scar	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Skin and subcutaneous tissue disorders Papule	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Skin and subcutaneous tissue disorders Rash	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Skin and subcutaneous tissue disorders Rash Papular	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Skin and subcutaneous tissue disorders Rash Pruritic	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Skin and subcutaneous tissue disorders Skin Irritation	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Skin and subcutaneous tissue disorders Urticaria	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.
Vascular disorders Hypotension	12.5% 1/8 • From first dose of study drug until until 30 days after the last dose of study drug (treatment emergent events). Mean (SD) treatment duration was 98.11 (29.02) weeks.

Additional Information

Medical Information

Ultragenyx Pharmaceutical Inc

Phone: 1-888-756-8567

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
Publication restrictions are in place

Restriction type: OTHER