Clinical Trial

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

Completed Phase 2
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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.
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2025-04-22.
Trial Details
NCT Number NCT06949358
Lead Sponsor Sanofi
Conditions Acid Sphingomyelinase Deficiency
Enrollment 3 participants
Start Date 2021-11-18
Primary Completion 2025-04-08 (estimated)
Study Completion 2025-04-08 (estimated)
Updated on ClinicalTrials.gov 2026-04-13