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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
Sponsor: Sanofi
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.
Official title: 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
Key Details
Gender
All
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
3
Start Date
2021-11-18
Completion Date
2025-04-08
Last Updated
2026-04-13
Healthy Volunteers
No
Conditions
Interventions
Olipudase alfa
Pharmaceutical form:Powder for concentrate for solution for infusion-Route of administration:intravenous infusion
Locations (2)
Investigational Site Number : 2500002
Bron, France
Investigational Site Number : 2500001
Paris, France