Clinical Trial: Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional
Official Title: A Phase IIb, Open-label Study to Assess the Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of Multiple Subcutaneous Doses of PRO045 in Subjects With Duchenne Muscular Dy
Brief Summary: The purpose of the study is to see whether PRO045 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 45 in the DNA for the dystrophin protein.
Detailed Summary: A phase IIb, open-label, multiple-dose study. The study consists of two phases; a dose escalation phase (with subsequent dose-titration) and a 48-week treatment phase.
Sponsor: BioMarin Pharmaceutical
Current Primary Outcome: Change from baseline in 6 minute walk test [ Time Frame: after 48 weeks of treatment phase ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Muscle function [ Time Frame: after 48 weeks of treatment phase ]
- Muscle strength [ Time Frame: after 48 weeks treatment phase ]
- Performance of upper limb [ Time Frame: after 48 weeks of treatment phase ]
- Functional outcomes questionnaire [ Time Frame: after 48 weeks of treatment ]
- Safety [ Time Frame: after 48 weeks of treatment phase ]
Original Secondary Outcome: Same as current
Information By: BioMarin Pharmaceutical
Dates:
Date Received: March 20, 2013
Date Started: January 2013
Date Completion: July 2017
Last Updated: October 27, 2016
Last Verified: June 2015
