Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy

Completed · Phase 1

Conditions studied: Duchenne Muscular Dystrophy

In brief

The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).

Key facts

Study ID

NCT00428935

Run by

Nationwide Children's Hospital

People needed

6

Starts

2006-03-01

Expected to finish

2010-07-01

Last updated by the study team

2013-02-05

Who can join

Age: 5 and older, up to 15. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Known null mutation of the Dystrophin gene
• Male age of 5 years or older
• If taking corticosteroids, must have dose unchanged for the past 3 months
• Serum creatine kinase elevation greater than 10x normal value (established by Children's Hospital)
• Progressive, symmetrical proximal muscle weakness of arms and legs

You may not qualify if…

• Unable to cooperate for muscle strength testing
• Joint contractures that prohibit muscle strength testing
• Concomitant illness
• Individuals predisposed to excessive vagal responses (bradyarrhythmia or hypotension)
• Controlled substance abuse

Where it is running

• Columbus Children's Hospital — Columbus, Ohio, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.