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Duchenne Muscular DystrophyPhase 1NCT06114056

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

A Phase 1 study testing JWK007 Single intravenous infusion administration in people with Duchenne Muscular Dystrophy, run by West China Hospital at 1 site in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

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). Read more about this study

Who is it for?
Ages 5 to 10 years, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
JWK007 Single intravenous infusion administration
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
3 participants
Where
1 siteChina
Study dates
31 Jan 2024to 13 Nov 2029
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 to 10 years
  • Men only
  • No healthy volunteers
  • Child

Who may be able to join

  • Participants meeting all of the following criteria may be considered for inclusion:
  • Male, aged 5 to 10 years (inclusive).
  • 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.
  • Below-average performance on motor assessment testing.
  • Ability to cooperate with motor assessment testing.
  • Tolerance for muscle biopsy under anesthesia with no contraindications for biopsy.
  • 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.

Who cannot join

  • Participants meeting any one of the following criteria are not eligible for inclusion:
  • Active viral infection based on clinical observations.
  • Signs of cardiomyopathy, including echocardiogram with ejection fraction below 40%.
  • Serological evidence of HIV infection, or Hepatitis B or C infection.
  • Diagnosis of (or ongoing treatment for) an autoimmune disease.
  • 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).
Show all 13Show fewer
  • Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer.
  • Subjects with AAVM101 neutralizing antibody titers > 1:400 as determined by ELISA immunoassay.
  • 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.
  • Severe infection (eg. pneumonia, pyelonephritis, or meningitis) within 4 weeks before gene transfer visit (enrollment may be postponed).
  • 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.
  • Has had any type of gene therapy, cell based therapy (eg. stem cell transplantation), or CRISPR/Cas9.
  • Family does not want to disclose patient's study participation with primary care physician and other medical providers

Trial locations and contacts

1 site in 1 country.

China1 site

  • West China Hospital, Sichuan University

    Chengdu, Sichuan, 610041

Other trials in China

About this study

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).

Read the 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.

Being tested
JWK007 Single intravenous infusion administration
Keywords
Duchenne Muscular Dystrophy, Gene Therapy, Adeno-associated virus
Medical subject headings
Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • JWK007 Single intravenous infusion administrationBiological

    Each patient receives a single intravenous infusion of JWK007 at a dose of 1.0 × 10^14 vg/kg.

Study groups in NCT06114056
Study groupRoleWhat they receive
JWK007 (AAVM101-µDys)ExperimentalJWK007 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.
Receives: Biological: JWK007 Single intravenous infusion administration

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. adverse events

    Adverse events defined as the number of participants with adverse events according CTCAE v5.0

    Measured: 5 years

Secondary outcomes (5)

  1. North Star Ambulatory Assessment

    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.

    Measured: 5 years

  2. Six-Minute Walk Test

    the distance the patient walked in six minutes

    Measured: 5 years

  3. 10-Meter Walk/Run Test

    The time it takes to walk 100 meters

    Measured: 5 years

  4. creatine kinase

    Changes in circulating levels of CK

    Measured: 5 years

  5. the expression of micro-dystrophin gene

    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.

    Measured: 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Nov 2023
  2. Study start31 Jan 2024
  3. Primary completion13 Nov 2029 (estimated)
  4. Study completion13 Nov 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
3 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06114056
Sponsor's protocol ID
2023-1285
Status last verified
Feb 2026
First submitted
29 Oct 2023
Last updated
10 Sep 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Costa Verdera H, Kuranda K, Mingozzi F. AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer. Mol Ther. 2020 Mar 4;28(3):723-746. doi: 10.1016/j.ymthe.2019.12.010. Epub 2020 Jan 10.

    PubMed 31972133

  • Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Sep 1;77(9):1122-1131. doi: 10.1001/jamaneurol.2020.1484.

    PubMed 32539076

  • Miesbach W, Meijer K, Coppens M, Kampmann P, Klamroth R, Schutgens R, Tangelder M, Castaman G, Schwable J, Bonig H, Seifried E, Cattaneo F, Meyer C, Leebeek FWG. Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B. Blood. 2018 Mar 1;131(9):1022-1031. doi: 10.1182/blood-2017-09-804419. Epub 2017 Dec 15.

    PubMed 29246900

  • Duan D. Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy. Mol Ther. 2018 Oct 3;26(10):2337-2356. doi: 10.1016/j.ymthe.2018.07.011. Epub 2018 Jul 17.

    PubMed 30093306

  • Xue F, Li H, Wu X, Liu W, Zhang F, Tang D, Chen Y, Wang W, Chi Y, Zheng J, Du Z, Jiang W, Zhong C, Wei J, Zhu P, Fu R, Liu X, Chen L, Pei X, Sun J, Cheng T, Yang R, Xiao X, Zhang L. Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial. Lancet Haematol. 2022 Jul;9(7):e504-e513. doi: 10.1016/S2352-3026(22)00113-2. Epub 2022 May 19.

    PubMed 35598604

  • Roberts TC, Wood MJA, Davies KE. Therapeutic approaches for Duchenne muscular dystrophy. Nat Rev Drug Discov. 2023 Nov;22(11):917-934. doi: 10.1038/s41573-023-00775-6. Epub 2023 Aug 31.

    PubMed 37652974

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing JWK007 Single intravenous infusion administration in people with Duchenne Muscular Dystrophy, run by West China Hospital at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 10 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

adverse events (time frame: 5 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06114056, which also lists 6 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2026.

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