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A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004704 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2024-07-03

Study results available
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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.

Conditions

  • Sphingomyelin Lipidosis

Interventions

DRUG

GZ402665

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

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-12-04
Primary Completion
2023-09-06
Completion
2023-09-06

Countries

  • United States
  • Belgium
  • Brazil
  • France
  • Germany
  • Italy
  • United Kingdom

Study Locations

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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 NCT02004704 on ClinicalTrials.gov