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.