At a glance
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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
In Brief
A Phase 2 clinical trial evaluating Olipudase alfa for Acid Sphingomyelinase Deficiency. Completed, enrolled 3 participants across 2 sites.
Detailed 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.
Study Details
Timeline
Interventions
Pharmaceutical form:Powder for concentrate for solution for infusion-Route of administration:intravenous infusion