Trial Outcomes & Findings for Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency (NCT NCT01307098)

Last Updated: 2018-12-11

Results Overview

Safety and tolerability of sebelipase alfa was primarily assessed by monitoring the number of participants reporting treatment-emergent adverse events (TEAEs), including serious adverse events, and infusion-related reactions (IRRs). The number of participants who discontinued from the study due to a TEAE is also presented. An IRR was defined as any adverse event that occurred between the start of the infusion and 4 hours after completion of the infusion and was assessed by the Investigator as at least possibly related to study drug. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

9 participants

Primary outcome timeframe

Screening up to Day 52

Results posted on

2018-12-11

Participant Flow

Participants were screened for eligibility for enrollment in this study (LAL-CL01). Nine participants met all enrollment criteria and were enrolled and allocated to 1 of 3 cohorts.

Participant milestones

Participant milestones
Measure	Sebelipase Alfa 0.35 mg/kg Cohort 1: Participants were administered once weekly (qw) infusions of 0.35 milligrams/kilogram (mg/kg) sebelipase alfa.	Sebelipase Alfa 1 mg/kg Cohort 2: Participants were administered qw infusions of 1 mg/kg sebelipase alfa.	Sebelipase Alfa 3 mg/kg Cohort 3: Participants were administered qw infusions of 3 mg/kg sebelipase alfa.
Overall Study STARTED	3	3	3
Overall Study Received at Least 1 Dose of Study Drug	3	3	3
Overall Study COMPLETED	3	3	3
Overall Study NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

Baseline characteristics by cohort

PRIMARY outcome

Timeframe: Screening up to Day 52

Population: Safety Analysis Set: All participants who received any full or partial dose of sebelipase alfa in this study.

Outcome measures

Outcome measures
Measure	Sebelipase Alfa 0.35 mg/kg n=3 Participants Cohort 1: Participants were administered qw infusions of 0.35 mg/kg sebelipase alfa.	Sebelipase Alfa 1 mg/kg n=3 Participants Cohort 2: Participants were administered qw infusions of 1 mg/kg sebelipase alfa.	Sebelipase Alfa 3 mg/kg n=3 Participants Cohort 3: Participants were administered qw infusions of 3 mg/kg sebelipase alfa.
Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) TEAEs	1 participants	3 participants	3 participants
Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) SAEs	0 participants	0 participants	0 participants
Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) IRRs	0 participants	0 participants	0 participants
Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) TEAEs Leading to Study Discontinuation	0 participants	0 participants	0 participants

Adverse Events

Sebelipase Alfa 0.35 mg/kg

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Sebelipase Alfa 1 mg/kg

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Sebelipase Alfa 3 mg/kg

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Alexion Pharmaceuticals Inc.

Phone: 855-752-2356

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Sebelipase Alfa 0.35 mg/kg n=3 Participants Cohort 1: Participants were administered qw infusions of 0.35 mg/kg sebelipase alfa.	Sebelipase Alfa 1 mg/kg n=3 Participants Cohort 2: Participants were administered qw infusions of 1 mg/kg sebelipase alfa.	Sebelipase Alfa 3 mg/kg n=3 Participants Cohort 3: Participants were administered qw infusions of 3 mg/kg sebelipase alfa.	Total n=9 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	9 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Continuous	32.3 years STANDARD_DEVIATION 7.57 • n=5 Participants	35.3 years STANDARD_DEVIATION 14.22 • n=7 Participants	27.0 years STANDARD_DEVIATION 12.17 • n=5 Participants	31.6 years STANDARD_DEVIATION 10.74 • n=4 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	3 Participants n=4 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants	6 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	9 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 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	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	9 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants

Measure	Sebelipase Alfa 0.35 mg/kg n=3 participants at risk Cohort 1: Participants were administered qw infusions of 0.35 mg/kg sebelipase alfa.	Sebelipase Alfa 1 mg/kg n=3 participants at risk Cohort 2: Participants were administered qw infusions of 1 mg/kg sebelipase alfa.	Sebelipase Alfa 3 mg/kg n=3 participants at risk Cohort 3: Participants were administered qw infusions of 3 mg/kg sebelipase alfa.
Gastrointestinal disorders Abdominal discomfort	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Gastrointestinal disorders Abdominal distension	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Gastrointestinal disorders Abdominal pain	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Gastrointestinal disorders Abdominal pain lower	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Gastrointestinal disorders Diarrhoea	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	66.7% 2/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Gastrointestinal disorders Dyspepsia	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Gastrointestinal disorders Flatulence	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Gastrointestinal disorders Nausea	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Gastrointestinal disorders Rectal haemorrhage	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
General disorders Catheter site related reaction	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
General disorders Feeling hot	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
General disorders Oedema peripheral	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Infections and infestations Upper respiratory tract infection	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Infections and infestations Viral upper respiratory tract infection	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Metabolism and nutrition disorders Hypercholesterolaemia	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Metabolism and nutrition disorders Hypertriglyceridaemia	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Musculoskeletal and connective tissue disorders Bone pain	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Musculoskeletal and connective tissue disorders Myalgia	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Nervous system disorders Headache	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	66.7% 2/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Nervous system disorders Presyncope	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Nervous system disorders Syncope	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Renal and urinary disorders Urine odour abnormal	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Reproductive system and breast disorders Breast swelling	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Reproductive system and breast disorders Vaginal haemorrhage	0.00% 0/1 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	100.0% 1/1 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/1 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Skin and subcutaneous tissue disorders Dermatitis contact	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.
Skin and subcutaneous tissue disorders Night sweats	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	0.00% 0/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.	33.3% 1/3 • Screening up to Day 52 Adverse events were obtained through participant reporting or were elicited by specific questioning or examination of the participant.