NCT01722526

Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients

An Open-label, Multicenter, Ascending Dose Study of the Tolerability and Safety of Recombinant Human Acid Sphingomyelinase (rhASM) in Patients With Acid Sphingomyelinase Deficiency (ASMD)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of rhASM in adult patients with Acid Sphingomyelinase Deficiency (ASMD) following repeated-dose administration.

Conditions

• Human Acid Sphingomyelinase Deficiency

Interventions

Type	Name	Description
DRUG	Recombinant human acid sphingomyelinase	Administered intravenously every 2 weeks for 26 weeks

Timeline

• Start date: 2013-03-01
• Primary completion: 2014-01-01
• Completion: 2014-01-01
• First posted: 2012-11-07
• Last updated: 2015-07-30

Locations

2 sites across 2 countries: United States, United Kingdom