NCT02004704

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa. The secondary objectives of this study were to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration.

Detailed description

LTS13632 is a multicenter, nonrandomized, open-label, long-term extension study of participants with ASMD who have previously participated in a study of olipudase alfa. (DFI13803 for pediatric participants and DFI13412 for adult participants). The maximum study duration per participant was 9 years or until olipudase alfa becomes commercially accessible. Notwithstanding the above, every pediatric participant were treated in LTS13632 study for at least 3 years to comply with the requirements agreed in the olipudase alfa Pediatric Investigational Plan.

Conditions

• Sphingomyelin Lipidosis

Interventions

Type	Name	Description
DRUG	GZ402665	Pharmaceutical form: Powder for concentrate for solution for infusion Route of administration: intravenous infusion

Timeline

• Start date: 2013-12-04
• Primary completion: 2023-09-06
• Completion: 2023-09-06
• First posted: 2013-12-09
• Last updated: 2024-07-03
• Results posted: 2024-07-03

Locations

9 sites across 7 countries: United States, Belgium, Brazil, France, Germany, Italy, United Kingdom