CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 9 enrolled
Drug / intervention
Sebelipase alfa (SBC-102)drug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT01371825
NCT01371825Phase 3Completed
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An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Alexion Pharmaceuticals, Inc.·interventional·Posted Jun 13, 2011·Updated Jan 30, 2019

In Brief

A Phase 3 clinical trial evaluating Sebelipase alfa (SBC-102) for Lysosomal Acid Lipase Deficiency and Wolman Disease. Completed, enrolled 9 participants across 7 sites in 5 countries.

Detailed Summary

This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency. Eligible participants received once-weekly (qw) infusions of sebelipase alfa for up to 5 years.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsLysosomal Acid Lipase Deficiency, Wolman Disease
CountriesEgypt, France, Ireland, United Kingdom, United States
Collaborators--

Timeline

Phase 3CompletedFinished
2011201220132014201520162017201820192020202120222023202420252026
First PostedJun 13, 2011
Enrollment StartMay 4, 2011
Primary CompletionJan 3, 2018
TodayJul 2, 2026
Enrollment to primary: 6.7 yearsPosted 15.1 years ago

Interventions

Sebelipase alfa (SBC-102)drug

Sebelipase alfa is a recombinant human lysosomal acid lipase enzyme. The investigational medicinal product is an enzyme replacement therapy intended for treatment of participants with LAL Deficiency. Dosing occurred qw for up to 5 years.