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Clinical Trial Report

Gene Therapy Trial Report

Summary

An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)


NCTID NCT06594094 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Duchenne Muscular Dystrophin (DMD)
Disease Ontology Term DOID:11723
Compound Name HG302
Sponsor HuidaGene Therapeutics Co., Ltd.
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 6
Results Posted Not Available

Therapy Information


Target Gene/Variant DMD (exon 51 splice site)
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Exon skipping/splice editor
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV
Editor Type hfCas12Max
Dose 1 Up to 2 dose cohorts, undisclosed concentration
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Na
Submit Date 2024-09-10
Completion Date 2026-09-30
Last Update 2024-11-25

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 First patient dosed announced in December 2024

Resources/Links