Overview

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

Status:
Completed
Trial end date:
2017-12-28
Target enrollment:
0
Participant gender:
All
Summary
This study evaluated the safety and efficacy of sebelipase alfa in a broad population of participants with lysosomal acid lipase deficiency (LAL-D).
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Alexion Pharmaceuticals
Criteria
Key Inclusion Criteria:

1. Participant was >8 months of age at the time of dosing.

2. 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.

3. 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

4. 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:

1. Participant had known causes of active liver disease other than LAL-D, which had not
been adequately treated.

2. Participant received a hematopoietic stem cell or liver transplant <2 years from the
time of dosing.

3. 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.