Phase 2
N=31
Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
Lysosomal Acid Lipase Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT02112994 ↗Enrolled (actual)
31
Serious AEs
22.5%
Results posted
Jan 2019
Primary outcome: Primary: Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs) — 1; 1; 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Sebelipase Alfa (Drug)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Dec 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs) |
1; 1; 2 | — |
| SECONDARY Percent Change In Serum Lipids From Baseline To Week 144 |
-37.5; -29.2; -22.5; 76.5; 24.2; 6.1 | — |
| SECONDARY Participants Testing Positive For Anti-drug Antibodies (ADAs) |
2; 0 | — |
| SECONDARY Percent Change In Body Mass Index (BMI)-For-Age Percentile From Baseline To Week 144 In Pediatric Participants |
26.45 | — |
| SECONDARY Shift In Child-Pugh Status From Baseline To Week 144 |
16; 1; 1 | — |
Summary
This study evaluated the safety and efficacy of sebelipase alfa in a broad population of participants with lysosomal acid lipase deficiency (LAL-D).
Eligibility Criteria
Key Inclusion Criteria
- Participant was >8 months of age at the time of dosing.
- Confirmation of LAL-D diagnosis as determined by the central laboratory or, for participants with prior hematopoietic stem cell transplant or liver transplant, historical enzyme activity or molecular genetic testing confirming a diagnosis of LAL-D.
- Participants >8 months but <4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:
- Dyslipidemia
- Elevated transaminases
- Impaired growth
- Suspected malabsorption
- Other clinical manifestation of LAL-D
- Participants ≥4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:
- Evidence of advanced liver disease
- Histologically confirmed disease recurrence in participants with past liver or hematopoietic transplant
- Persistent dyslipidemia
- Suspected malabsorption
- Other clinical manifestation of LAL-D
Key Exclusion Criteria
- Participant had known causes of active liver disease other than LAL-D, which had not been adequately treated.
- Participant received a hematopoietic stem cell or liver transplant <2 years from the time of dosing.
- Participant with co-morbidities other than complications due to LAL-D, which were irreversible or associated with a high mortality risk within 6 months or would interfere with study compliance or data interpretation.
Data sourced from ClinicalTrials.gov (NCT02112994). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.