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

March 15, 2026 updated by: Xingchen Peng, West China Hospital
This study is a single-center, single-arm, non-randomized, open-label, non-controlled, dose-escalation, prospective clinical trial designed to assess the safety, tolerability, and preliminary efficacy of JWK007 injection in pediatric patients with Duchenne Muscular Dystrophy (DMD).

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

DMD is a rare genetic disorder that primarily affects males. This disease is closely associated with mutations in the DMD gene located on the X chromosome. The DMD gene encodes a protein known as dystrophin, which plays a crucial role in providing essential structural and protective support within the muscles. Gene therapy drugs using Adeno-Associated Virus (AAV) as a vector hold the promise of offering a convenient, effective, and safe treatment option for DMD patients. Therefore, we have independently developed and designed the JWK007 injection. The study was originally designed as a '3+3' dose-escalation trial with two cohorts. However, due to recruitment difficulties at the higher dose level, only the low-dose cohort (N=3) will be enrolled. No further enrollment or dose escalation will be conducted, and all safety, tolerability, and preliminary efficacy endpoints will be assessed in this single low-dose cohort.

Study Type

Interventional

Enrollment (Actual)

3

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Sichuan
      • Chengdu, Sichuan, China, 610041
        • West China Hospital, Sichuan University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: JWK007 (AAVM101-µDys)
JWK007 injection utilizes AAVM101, an AAVrh74-derived capsid engineered for enhanced muscle targeting, as its viral vector. Because the full-length dystrophin gene exceeds the conventional rAAV packaging capacity (< 5.0 kb), JWK007 delivers an independently designed micro-dystrophin (μDystrophin) gene. The resulting μDystrophin protein retains essential functional domains, including structures critical for promoting neuronal nitric oxide synthase (nNOS) activity and membrane binding.
Biological: JWK007 Single intravenous infusion administration
Each patient receives a single intravenous infusion of JWK007 at a dose of 1.0 × 10^14 vg/kg.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
adverse events
Time Frame: 5 years
Adverse events defined as the number of participants with adverse events according CTCAE v5.0
5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
North Star Ambulatory Assessment
Time Frame: 5 years
North Star Ambulatory Assessment (NSAA) is a clinical tool used to assess the motor function and ambulatory capabilities of children and adolescents with neuromuscular disorders like Duchenne muscular dystrophy.
5 years
Six-Minute Walk Test
Time Frame: 5 years
the distance the patient walked in six minutes
5 years
10-Meter Walk/Run Test
Time Frame: 5 years
The time it takes to walk 100 meters
5 years
creatine kinase
Time Frame: 5 years
Changes in circulating levels of CK
5 years
the expression of micro-dystrophin gene
Time Frame: 6 months
Baseline muscle biopsies for dystrophin expression will be performed between -30 and -7 days prior to treatment in all subjects. All subjects will undergo a post-treatment biopsy on day 180. Micro-dystrophin gene expression was quantified (immunofluorescence and Western blot analysis) and compared before and after muscle biopsy.
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

West China Hospital

Investigators

  • Principal Investigator: Xingchen Xingchen, Ph.D, West China Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 31, 2024

Primary Completion (Estimated)

November 13, 2029

Study Completion (Estimated)

November 13, 2029

Study Registration Dates

First Submitted

October 29, 2023

First Submitted That Met QC Criteria

October 29, 2023

First Posted (Actual)

November 2, 2023

Study Record Updates

Last Update Posted (Actual)

March 17, 2026

Last Update Submitted That Met QC Criteria

March 15, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2023-1285

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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