NCT06949358

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

A Long-Term Follow-up Study to Evaluate Safety and Tolerability of Olipudase Alfa in Patients Who Completed the DFI12712 or the LTS13632 Study in France

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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

Type	Name	Description
DRUG	Olipudase alfa	Pharmaceutical form:Powder for concentrate for solution for infusion-Route of administration:intravenous infusion

Timeline

• Start date: 2021-11-18
• Primary completion: 2025-04-08
• Completion: 2025-04-08
• First posted: 2025-04-29
• Last updated: 2026-04-13
• Results posted: 2026-04-13

Locations

2 sites across 1 country: France

Regulatory

• FDA-regulated drug study