CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 9 enrolled
Drug / intervention
Sebelipase alfa 0.35 mg/kg +2 moredrug
Likely dose
Sebelipase alfa 0.35 mg/kgfrom 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/NCT01307098
NCT01307098Phase 2Completed
CompareCT.gov

An Open-Label Multicenter Study to Evaluate the Safety, Tolerability and Pharmacokinetics of SBC-102 in Adult Participants With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency

Alexion Pharmaceuticals, Inc.·interventional·Posted Mar 2, 2011·Updated Dec 11, 2018

In Brief

A Phase 2 clinical trial evaluating Sebelipase alfa 0.35 mg/kg, Sebelipase alfa 1 mg/kg, and 1 other intervention for Cholesterol Ester Storage Disease(CESD) and 2 related conditions. Completed, enrolled 9 participants across 7 sites in 4 countries.

Detailed Summary

This was the first clinical study of SBC-102 (sebelipase alfa) for the treatment of Lysosomal Acid Lipase (LAL) Deficiency. It was an open-label dose escalation study in adult participants with liver dysfunction due to LAL Deficiency and was designed to examine 3 doses of sebelipase alfa. The targeted number for this study was 9 evaluable participants.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsCholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
CountriesCzechia, France, United Kingdom, United States
Collaborators--

Timeline

Phase 2CompletedFinished
2011201220132014201520162017201820192020202120222023202420252026
First PostedMar 2, 2011
Enrollment StartApr 25, 2011
Primary CompletionJan 6, 2012
TodayJul 2, 2026
Enrollment to primary: 8 monthsPosted 15.3 years ago

Interventions

Sebelipase alfa 0.35 mg/kgdrug

Sebelipase alfa is a recombinant human lysosomal acid lipase.

Sebelipase alfa 1 mg/kgdrug

Sebelipase alfa is a recombinant human lysosomal acid lipase.

Sebelipase alfa 3 mg/kgdrug

Sebelipase alfa is a recombinant human lysosomal acid lipase.