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Clinical Trial Report

Gene Therapy Trial Report

Summary

Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy

NCTID NCT06641895 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term DOID:11723
Compound Name BBM-D101
Sponsor Shanghai Jiao Tong University School of Medicine
Funder Type Other
Recruitment Status
Recruiting
Enrollment Count 6
Results Posted Not Available

Therapy Information

Target Gene/Variant Undisclosed
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Undisclosed
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell Undisclosed
Delivery System Viral transduction
Vector Type AAV
Editor Type none
Dose 1 Undisclosed dose
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Early phase1
Submit Date 2024-10-08
Completion Date 2030-07-31
Last Update 2025-03-25

Participation Criteria

Eligible Age 4 Years - 8 Years
Standard Ages Child
Sexes Eligible for Study MALE

Locations

No.of Trial Sites 1
Locations China

Regulatory Information

Has US IND False
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates IND cleared January 2025

Resources/Links

News and Press Releases
Protocol
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