NCT05359276

Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France

Acid Sphingomyelinase Deficiency (ASMD): Data Analysis of Adult and Pediatric Patients on Early Access to Olipudase Alfa in France

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 40 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

Primary Objective: To describe the lung, spleen and liver outcomes of olipudase alfa Secondary Objectives: * To describe the patient's characteristics * To describe conditions of olipudase alfa use * To describe safety data related to the use of olipudase alfa * To describe complementary effectiveness outcomes parameters

Detailed description

Approximate duration of enrollment: 30 months Total study duration: approximately 30 months This is a national, multicenter observational retrospective and prospective cohort data collection study. Retrospective is defined as collection of data from all patients, including deceased patients, who were already on early access olipudase alfa in France before the start of this study.

Conditions

• Acid Sphingomyelinase Deficiency (ASMD)

Interventions

Type	Name	Description
DRUG	Olipudase alfa	GZ402665

Timeline

• Start date: 2022-06-10
• Primary completion: 2024-12-31
• Completion: 2024-12-31
• First posted: 2022-05-03
• Last updated: 2025-02-06

Locations

1 site across 1 country: France