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Gene Therapy Trial Browser
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
The purpose of this trial is to study the evaluation of clinical safety and feasibility of gene therapy in patients with limb girdle muscular dystrophy type 2C (gamma-sarcoglycanopathy).
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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
Clinical Publications
A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C