CompletedPhase 2

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

United States, Australia, Belgium31 participantsStarted 2014-06-24

Plain-language summary

This study evaluated the safety and efficacy of sebelipase alfa in a broad population of participants with lysosomal acid lipase deficiency (LAL-D).

Age range8 Months

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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:
✓. Participants ≥4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:

✕. 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.

Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)

Timeframe: Screening, Week 144

NCT IDNCT02112994

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-12-28

Results submitted2018-12-26