Understanding the safety of CRISPR gene-editing therapy in boys with Duchenne muscular dystrophy

An Investigator-initiated Clinical Study Evaluating the CRISPR-hfCas12Max Gene Editing Therapy in the Treatment of Duchenne Muscular Dystrophy (DMD)

This study is testing a new CRISPR gene-editing therapy to see if it is safe and can improve muscle function in young boys with Duchenne muscular dystrophy.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 4 Years to 8 Years
• Sex: Male
• Sponsor: HuidaGene Therapeutics Co., Ltd. (Industry-sponsored)
• Locations: 1 site (Shanghai, Shanghai)
• Trial ID: NCT06594094 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the safety and long-lasting effects of a CRISPR gene-editing therapy, HG302, in boys diagnosed with Duchenne muscular dystrophy (DMD). The study focuses on males aged 4 to 8 years with specific DMD gene mutations, aiming to assess the therapy's impact on muscle function and overall health. Participants will undergo a dose-escalation approach to determine the optimal dosage while monitoring for any adverse effects. The trial seeks to provide a potential breakthrough in treating a condition that currently has limited therapeutic options.

Who should consider this trial

Good fit: Ideal candidates are males aged 4 to 8 years with specific DMD gene mutations and the ability to walk independently.

Not a fit: Patients with active infections, severe respiratory issues, or prior gene therapy treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly improve muscle function and quality of life for boys with DMD.

How similar studies have performed: While gene-editing therapies are a novel approach, similar studies have shown promise in other genetic disorders, indicating potential for success in this area.

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.

Where this trial is running

Shanghai, Shanghai

• Shanghai Children s Medical Center Affiliated to Shanghai Jiao Tong University School of Medical — Shanghai, Shanghai, China (Recruiting)

Study contacts

• Study coordinator: Study Director

Conditions Duchenne Muscular Dystrophin; DMD; dystrophin; Gene-editing; HG302; CRISPR