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)

Plain-language summary

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.

Trial details

Phase

Early Phase 1

Status

Completed

Sponsor

HuidaGene Therapeutics Co., Ltd.

Intervention

HG302

Start date

11/6/2024

Estimated completion

12/2/2025

Countries

China

Related genes

Locations

• Shanghai, China

Eligibility criteria

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.