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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 Dystrophy (DMD)
Disease Ontology Term DOID:11723
Compound Name HG302
Sponsor HuidaGene Therapeutics Co., Ltd.
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 4 (ACTUAL)
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
Delivery System Viral transduction
Vector Type AAV
Editor Type hfCas12Max
Dose 1 Up to 2 dose cohorts, undisclosed concentration

Study Record Dates


Current Stage Early phase1
Submit Date 2024-09-10
Completion Date 2025-12-02
Last Update 2026-02-17

Participation Criteria


Eligible Age 4 Years - 8 Years
Standard Ages Child
Sexes Eligible for Study MALE
Eligibility Criteria
Inclusion Criteria: * Males ≥ 4 and ≤8 years at the time of signing informed consent, with clinical diagnosis of DMD; * DMD gene mutation types are deletions in exons 52, 52-61, or 52-63; * Able to walk at least 10 meters independently; * Willing to cooperate with muscle biopsy test; * Acceptable hematology, clinical chemistry, and urine laboratory parameters. Exclusion Criteria: * Presence of active infection; * Presence of DMD-associated cardiomyopathy manifestations; * Respiratory insufficiency requiring invasive or non-invasive ventilation; * Serious infections such as pneumonia, pyelonephritis, or meningitis within 4 weeks prior to receiving trial drug infusion; * Prior central nervous system surgery within 6 months before enrolment; * Use of any investigational drug, or exon-skipping drug (whether investigational or not) 6 months prior to Screening; * Previous treatment with any gene therapy or cell therapy (e.g., stem cell transplantation); * Any other conditions that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

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