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

NCTID	NCT00428935 (View at clinicaltrials.gov)
Description	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). (Show More)
Development Status	Inactive
Indication	Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term	DOID:11723
Compound Name	RAAV2.5-CMV-mini-Dystrophin
Sponsor	Nationwide Children's Hospital
Funder Type	Other
Recruitment Status	Completed
Enrollment Count	6 (ACTUAL)
Results Posted	Not Available

Target Gene/Variant	Micro-dystrophin
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intramuscular (bicep)
Drug Product Type	Viral vector
Delivery System	Viral transduction
Vector Type	AAV2.5
Dose 1	6E11 vg
Dose 2	3.0E12 vg

Eligible Age	5 Years - 15 Years
Standard Ages	Child
Sexes Eligible for Study	MALE
Eligibility Criteria	Inclusion Criteria: * 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 Exclusion Criteria: * 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 View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Study drug was well tolerated by 6/6 subjects, limited efficacy: protein was detectable in 2/6 subjects at d43 post administration