Clinical Trial: A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Phase II Open-Label Clinical Trial of Recombinant Human Alpha-L-iduronidase (Aldurazyme®) to Evaluate the Safety and Pharmacokinetics in Mucopolysaccharidosis I (MPS I) Patients Less Than 5 Yea
Brief Summary: The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of enzyme replacement therapy with recombinant human alpha-L-iduronidase [Aldurazyme® (laronidase)] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy measurements will also be evaluated in this study.
Detailed Summary:
Sponsor: Genzyme, a Sanofi Company
Current Primary Outcome:
- Safety Evaluation [ Time Frame: 52 weeks ]Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.
- Pharmacokinetics - Area Under the (Plasma Concentration-time) Curve (AUC∞) [ Time Frame: 52 weeks ]AUC∞ is a measure of the total exposure to a drug.
- Pharmacokinetics - Elimination Half Life (t1/2) [ Time Frame: 52 weeks ]Half-life is the time it takes for the concentration of drug in plasma to decline by 50%.
- Pharmacokinetics - Total Plasma Clearance (CL) [ Time Frame: 52 weeks ]CL is volume of the body fluid cleared of the drug per unit of time.
- Pharmacokinetics - Volume of Distribution (Vz) [ Time Frame: 52 weeks ]Vz is the volume that relates the amount of drug in the body after absorption is complete to the concentration of drug in the plasma.
Original Primary Outcome: To evaluate the safety, PK and efficacy of Aldurazyme® (laronidase) in a patient population younger than 5 years old
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Sanofi
Dates:
Date Received: September 2, 2005
Date Started: October 2002
Date Completion:
Last Updated: March 17, 2015
Last Verified: March 2015
