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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
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

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