This study aims to understand the safety and long-lasting effects of HG302, a CRISPR gene-editing therapy, in patients with Duchenne muscular dystrophy (DMD). Boys aged 4-8 who can walk independently and who have deletions in exon 52, 52-61, or 52-63 of the DMD gene were able to take part. It was conducted in Early Phase 1 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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.