CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 25 enrolled
Drug / intervention
GZ402665drug
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/NCT02004704
NCT02004704Phase 2Completed
CompareCT.gov

A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Genzyme, a Sanofi Company·interventional·Posted Dec 9, 2013·Updated Jul 3, 2024

In Brief

A Phase 2 clinical trial evaluating GZ402665 for Sphingomyelin Lipidosis. Completed, enrolled 25 participants across 9 sites in 7 countries.

Detailed Summary

The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa. The secondary objectives of this study were to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSphingomyelin Lipidosis
CountriesBelgium, Brazil, France, Germany, Italy, United Kingdom, United States
Collaborators--

Timeline

Phase 2CompletedFinished
2014201520162017201820192020202120222023202420252026
First PostedDec 9, 2013
Enrollment StartDec 4, 2013
Primary CompletionSep 6, 2023
TodayJul 2, 2026
Enrollment to primary: 9.8 yearsPosted 12.6 years ago

Interventions

GZ402665drug

Pharmaceutical form: Powder for concentrate for solution for infusion Route of administration: intravenous infusion