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Clinical Trial Report
Gene Therapy Trial Report
Summary
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).
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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
Results Posted
Not Available
Therapy Information
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
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2.5
Editor Type
Dose 1
6E11 vg
Dose 2
3.0E12 vg
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2007-01-26
Completion Date
2010-07
Last Update
2013-02-05
Participation Criteria
Eligible Age
5 Years - 15 Years
Standard Ages
Child
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
1
Locations
United States
Regulatory Information
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
Resources/Links
Clinical Publications
Dystrophin immunity in Duchenne's muscular dystrophy
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector