Clinical Trial: Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: An Open-label, Multicenter, Ascending Dose Study of the Tolerability and Safety of Recombinant Human Acid Sphingomyelinase (rhASM) in Patients With Acid Sphingomyelinase Deficien
Brief Summary: To evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of rhASM in adult patients with Acid Sphingomyelinase Deficiency (ASMD) following repeated-dose administration.
Detailed Summary:
Sponsor: Genzyme, a Sanofi Company
Current Primary Outcome: Summary of Adverse Events (AEs) [ Time Frame: at least 26 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Pharmacokinetics as measured by peak plasma concentration (Cmax), time to peak concentration (tmax), area under curve (AUC), half life (t1/2), drug clearance (CL), and volume of distribution (Vss) [ Time Frame: up to 26 weeks ]
- Pharmacodynamics as measured by liver and skin biopsies, plasma, and dried blood spot [ Time Frame: up to 26 weeks ]
Original Secondary Outcome:
- • Pharmacokinetics as measured by peak plasma concentration (Cmax), time to peak concentration (tmax), area under curve (AUC), half life (t1/2), drug clearance (CL), and volume of distribution (Vss) [ Time Frame: up to 26 weeks ]
- • Pharmacodynamics as measured by liver and skin biopsies, plasma, and dried blood spot [ Time Frame: up to 26 weeks ]
Information By: Sanofi
Dates:
Date Received: November 5, 2012
Date Started: March 2013
Date Completion:
Last Updated: July 29, 2015
Last Verified: July 2015
