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Clinical Trial Report

Gene Therapy Trial Report

Summary

Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C


NCTID NCT01344798 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Limb-Girdle Muscular Dystrophy, Type 2C/R5
Disease Ontology Term DOID:0110277
Compound Name AAV1.DES.hSGCG
Sponsor Genethon
Funder Type Other
Recruitment Status
Completed
Enrollment Count 9
Results Posted Not Available

Therapy Information


Target Gene/Variant SGCG
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intramuscular (carpi radialis)
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV1
Editor Type
Dose 1 3E9 vg/100ul
Dose 2 1.5E10 vg/100ul
Dose 3 4.5E10 vg/300ul
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2011-03-17
Completion Date 2010-06
Last Update 2011-04-29

Participation Criteria


Eligible Age >=15 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations France

Regulatory Information


Has US IND
FDA Designations
Recent Updates Vector was well tolerated but expression of transgene was not detectable in most patients after 30 days

Resources/Links