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Gene Therapy Trial Report

Summary

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


NCTID NCT06114056 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term DOID:11723
Compound Name JWK007
Sponsor West China Hospital
Funder Type Other
Recruitment Status
Active not recruiting
Enrollment Count 3 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant Micro-dystrophin
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAVrh74
Editor Type none
Dose 1 1.0E14 vg/kg
Dose 2 2.0E14 vg/kg

Study Record Dates


Current Stage Phase1
Submit Date 2023-10-29
Completion Date 2029-11-13
Last Update 🔄 2026-03-17

Participation Criteria


Eligible Age 5 Years - 10 Years
Standard Ages Child
Sexes Eligible for Study MALE
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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates Investigator-initiated trial conducted at West China Hospital

Resources/Links


Resources/Links

No External Links Available.