Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)

NCTID	NCT00494195 (View at clinicaltrials.gov)
Description	Limb girdle muscular dystrophy type 2D (LGMD2D) is a genetic disease that affects skeletal muscle. Insufficient levels of the protein alpha-sarcoglycan result in muscle weakness that worsens over time. The purpose of this study is to evaluate the safety and effectiveness of gene therapy in treating children and adults with LGMD2D. (Show More)
Development Status	Inactive
Indication	Limb-Girdle Muscular Dystrophy, Type 2D/R3
Disease Ontology Term	DOID:0110278
Compound Name	RAAV1.tMCK.human-alpha-sarcoglycan
Sponsor	Nationwide Children's Hospital
Funder Type	Other
Recruitment Status	Completed
Enrollment Count	6
Results Posted	Not Available

Target Gene/Variant	SGCA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intramuscular (extensor digitorum brevis)
Drug Product Type	Viral vector
Target Tissue/Cell	Muscle
Delivery System	Viral transduction
Vector Type	AAV1
Editor Type
Dose 1	3.25E11 vg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Study drug was well tolerated by 3/3 subjects, protein was detectable in all subjects at 6 months post-injection. Clinical development of this program moved to Sarepta Therapeutics with optimized AAV capsid and modified intramuscular delivery