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A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France

NCT06949358 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2026-04-13

Study results available
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Summary

This was an open-label study to evaluate safety and tolerability and provide enzyme replacement therapy (ERT) with olipudase alfa to patients with acid sphingomyelinase deficiency (ASMD) who completed the DFI12712 or the LTS13632 Study in France until olipudase alfa reimbursement was granted in France.

Study and treatment duration:

The period between the participant's completion of Study DFI12712 or LTS13632 and olipudase alfa reimbursement was available in France.

In case reimbursement was not obtained, this study ended 5 years after starting.

Visit frequency: every 2 weeks.

Conditions

  • Acid Sphingomyelinase Deficiency

Interventions

DRUG

Olipudase alfa

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

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-11-18
Primary Completion
2025-04-08
Completion
2025-04-08
FDA Drug
Yes

Countries

  • France

Study Locations

More Related Trials

Entities

Companies

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 NCT06949358 on ClinicalTrials.gov