Clinical Trial: Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol
Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional
Official Title: A Continued Access Protocol for Eligible US Subjects With Duchenne Muscular Dystrophy Who Previously Participated in an Approved Drisapersen Study
Brief Summary: This is a single arm, open-label continued access protocol of drisapersen for the treatment of male subjects with Duchenne muscular dystrophy (DMD) having dystrophin mutations correctable by drisapersen-induced DMD Exon 51 skipping. The purpose of this continued access protocol is to offer pre-approval access to drisapersen for the treatment of subjects with DMD who previously participated in eligible drisapersen studies. The protocol will collect safety data required to assure subject safety and periodic efficacy data on muscle function.
Detailed Summary:
Sponsor: GlaxoSmithKline
Current Primary Outcome:
- Safety as assessed by the collection of adverse events (AEs) [ Time Frame: Baseline to Week 48 ]AEs will be collected from the start of Study Treatment and until 5 days post last-dose (at follow up).
- Safety as assessed by laboratory parameters [ Time Frame: Baseline to Week 48 ]Absolute values and changes over time of hematology, clinical chemistry, and urinalysis
- Safety as assesses by electrocardiogram (ECG) intervals [ Time Frame: Baseline to Week 48 ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
Original Secondary Outcome:
Information By: GlaxoSmithKline
Dates:
Date Received: June 27, 2013
Date Started: January 2014
Date Completion: July 2015
Last Updated: March 20, 2014
Last Verified: March 2014
