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An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency

NCT02376751 · Status: NO_LONGER_AVAILABLE · Type: EXPANDED_ACCESS

Last updated 2016-06-08

No results posted yet for this study

Summary

This is an open-label, multicenter expanded access protocol to allow patients with a confirmed diagnosis of Lysosomal Acid Lipase (LAL) Deficiency in the United States (US), access to sebelipase alfa (recombinant lysosomal acid lipase \[rhLAL\]) until commercial product is available.

Patients enrolled in the expanded access protocol will receive 1 mg/kg intravenous infusions of sebelipase alfa every other week.

Conditions

  • Lysosomal Acid Lipase Deficiency

Interventions

DRUG

sebelipase alfa

Sponsors & Collaborators

  • Alexion Pharmaceuticals, Inc.

    lead INDUSTRY

Eligibility

Min Age
8 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02376751 on ClinicalTrials.gov