Duchenne Muscular Dystrophy,DMD
Conditions
Interventions
Low-dose group (1.2×10^13 vg/kg):RG001 injection, single intravenous infusion, dose 1.2×10^13 vg/kg
High-dose group (6×10^13 vg/kg):RG001 injection, single intravenous infusion, dose 6×10^13 vg/kg
Sponsors
Chinese PLA General Hospital
Eligibility
Sex/Gender
Male
Age
4 Years to 8 Years
Inclusion criteria
Inclusion criteria: 1.Males aged 4–8 years at the time of study drug infusion, with a clinical and genetic diagnosis of DMD; 2.Ambulatory with a 6-minute walk test distance between 300 and 450 meters; 3.Able to cooperate with muscle biopsy and motor function tests; 4.Have been on a stable dose of glucocorticoids for at least 3 months prior to study drug administration; 5.Preexisting neutralizing antibody titer < 1:400.
Exclusion criteria
Exclusion criteria: 1. Presence of active infection (clinically consistent); 2. Presence of DMD-related cardiomyopathy manifestations, including: (1) Clinical symptoms or signs of cardiac function decline: such as exertional dyspnea, foot edema, orthopnea, basal lung crackles, etc.; (2) Imaging evidence of cardiac involvement: left ventricular ejection fraction (LVEF) measured by cardiac magnetic resonance (CMR) or transthoracic echocardiography (TTE) = 3 times the upper limit of normal (ULN); total bilirubin >= 1.5×ULN; creatinine >= 159 µmol/L; hemoglobin (Hb) 180 g/L; white blood cell count > 10.0 × 10^9/L; 9. Estimated glomerular filtration rate (eGFR) calculated by serum creatinine 1:400; 11. Presence of comorbidities or medical conditions requiring long-term medication that the investigator believes increases the risk of trial participation for the subject; 12. Serious infection within 4 weeks prior to study drug infusion, such as pneumonia, pyelonephritis, or meningitis; 13. Use of any investigational drug (except GC) within 6 months prior to screening, or exon-skipping drugs (whether investigational or not); 14. Previous gene therapy or cell therapy (e.g., stem cell transplantation); 15. Other conditions that the investigator believes may endanger the safety of the subject, affect adherence to the study, or interfere with the interpretation of study results.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse Events and Serious Adverse Events – Incidence and Severity; | — |
Secondary
| Measure | Time frame |
|---|---|
| 6-minute walk test,6MWT;North Star Ambulatory Assessment score;mini-utrophin expression; | — |
Countries
China
Contacts
Public ContactShiwen Wu
Chinese PLA General Hospital
Outcome results
None listed