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Clinical Trial Report

Gene Therapy Trial Report

Summary

Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis


NCTID NCT01519349 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis
Disease Ontology Term DOID:9883|DOID:3429
Compound Name RAAV1.CMV.huFollistatin344
Sponsor Nationwide Children's Hospital
Funder Type Other
Recruitment Status
Completed
Enrollment Count 15
Results Posted Not Available

Therapy Information


Target Gene/Variant FST (FS344)
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intramuscular
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV1
Editor Type
Dose 1 2E11 vg/kg (single leg)
Dose 2 3E11 vg/kg/quad
Dose 3 6E11 vg/kg/quad
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2012-01-23
Completion Date 2017-10
Last Update 2023-10-02

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Study drug was well tolerated, was not tested further

Resources/Links