NCT02292654

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Olipudase Alfa in Pediatric Patients Aged <18 Years With Acid Sphingomyelinase Deficiency

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 17 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks. Secondary Objective: To characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory efficacy of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.

Detailed description

The maximum study duration per participant was approximately 18 months (screening period: up to 60 days; treatment period: 64 weeks; post-treatment period: up to 37 days, not applicable if participants enrolled in a long-term extension treatment trial).

Conditions

• Sphingomyelin Lipidosis

Interventions

Type	Name	Description
DRUG	Olipudase alfa	Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous infusion

Timeline

• Start date: 2015-05-01
• Primary completion: 2019-12-09
• Completion: 2019-12-09
• First posted: 2014-11-17
• Last updated: 2022-03-23
• Results posted: 2021-02-01

Locations

6 sites across 6 countries: United States, Brazil, France, Germany, Italy, United Kingdom