Status:
RECRUITING
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)
Lead Sponsor:
HuidaGene Therapeutics Co., Ltd.
Conditions:
Duchenne Muscular Dystrophin (DMD)
Eligibility:
MALE
4-8 years
Phase:
NA
Brief Summary
Duchenne muscular dystrophin (DMD) is an X-linked, fatal muscle-wasting disease caused by mutations in the DMD gene encoding the dystrophin proteins, with symptom onset before age of 6 years in boys. ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
November 6 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 30 2026
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT06594094
Start Date
November 6 2024
End Date
September 30 2026
Last Update
November 25 2024
Active Locations (1)
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1
Shanghai Children s Medical Center Affiliated to Shanghai Jiao Tong University School of Medical
Shanghai, Shanghai Municipality, China