Evaluating JWK007 for treating Duchenne Muscular Dystrophy

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)

This study is testing a new treatment called JWK007 to see if it can help young boys with Duchenne Muscular Dystrophy feel better and improve their condition.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 5 Years to 10 Years
• Sex: Male
• Sponsor: West China Hospital (other)
• Locations: 1 site (Chengdu, Sichuan)
• Trial ID: NCT06114056 on ClinicalTrials.gov

What this trial studies

This clinical study assesses the safety, tolerability, and initial efficacy of a single intravenous infusion of JWK007 in pediatric patients diagnosed with Duchenne Muscular Dystrophy (DMD). It is a single-center, open-label, non-randomized trial that employs a dose-escalation design to incrementally increase dosages of the treatment. The study focuses on male participants aged 5 to 10 years with specific genetic mutations in the DMD gene, aiming to provide a new gene therapy option using Adeno-Associated Virus as a vector.

Who should consider this trial

Good fit: Ideal candidates for this study are male children aged 5 to 10 years with a confirmed diagnosis of Duchenne Muscular Dystrophy and specific genetic mutations.

Not a fit: Patients with active viral infections or those who do not meet the genetic and age criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could offer a novel therapeutic option for managing Duchenne Muscular Dystrophy, potentially improving muscle function and quality of life for affected children.

How similar studies have performed: While gene therapy approaches for DMD are being explored, this specific treatment with JWK007 represents a novel intervention that has not been previously tested.

Eligibility criteria

Inclusion Criteria:

Participants meeting all of the following criteria may be considered for inclusion:

1. Male, aged 5 to 10 years (inclusive).
2. Diagnosis of Duchenne Muscular Dystrophy (DMD) confirmed through medical history and genetic testing, characterized by a frameshift mutation (deletion or duplication) or a premature stop codon mutation in the DMD gene between exons 18 to 58.
3. Below-average performance on motor assessment testing.
4. Ability to cooperate with motor assessment testing.
5. Tolerance for muscle biopsy under anesthesia with no contraindications for biopsy.
6. Participants must have been taking a stable dose of oral corticosteroids for at least 12 weeks prior to screening, and the expected dose should remain constant throughout the study, except for adjustments related to changes in body weight.

Exclusion Criteria:

Participants meeting any one of the following criteria are not eligible for inclusion:

1. Active viral infection based on clinical observations.
2. Signs of cardiomyopathy, including echocardiogram with ejection fraction below 40%.
3. Serological evidence of HIV infection, or Hepatitis B or C infection.
4. Diagnosis of (or ongoing treatment for) an autoimmune disease.
5. Abnormal laboratory values considered clinically significant (GGT \> 3XULN, bilirubin ≥ 3.0 mg/dL, creatinine ≥ 1.8 mg/dL, Hgb \< 80 or \> 180 g/L; WBC \> 18.5\*10\^9/L).
6. Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer.
7. Subjects with AAVrh74 neutralizing antibody titers \> 1:400 as determined by ELISA immunoassay.
8. Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with the protocol required testing or procedures or compromise the subject's wellbeing, safety, or clinical interpretability.
9. Severe infection (eg. pneumonia, pyelonephritis, or meningitis) within 4 weeks before gene transfer visit (enrollment may be postponed).
10. Has received any investigational medication (other than corticosteroids) or exon skipping medications (including ExonDys 51), experimental or otherwise, in the last 6 months prior to screening for this study.
11. Has had any type of gene therapy, cell based therapy (eg. stem cell transplantation), or CRISPR/Cas9.
12. Family does not want to disclose patient's study participation with primary care physician and other medical providers

Where this trial is running

Chengdu, Sichuan

• West China Hospital, Sichuan University — Chengdu, Sichuan, China (RECRUITING)

Study contacts

• Principal investigator: Xingchen Xingchen, Ph.D — West China Hospital
• Study coordinator: Xingchen Peng, Ph.D

Conditions: Duchenne Muscular Dystrophy, Gene Therapy, Adeno-associated virus