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

• Status: COMPLETED
• Trial end date: 2025-04-08

Summary

This is 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 is granted in France.

Study and treatment duration:

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

In case reimbursement will not be obtained, this study will end 5 years after starting.

Visit frequency: every 2 weeks.

• Phase: PHASE2

Details

• Lead Sponsor: Sanofi
• Treatments: olipudase alfa