Duchenne/Becker muscular dystrophy
Conditions
Interventions
Vamorolone Treatment Group:None
Prednisone Treatment Group:None
Sponsors
West China Second Hospital, Sichuan University
Eligibility
Sex/Gender
Male
Age
4 Years to 18 Years
Inclusion criteria
Inclusion criteria: Only participants who meet **all** of the following criteria will be enrolled in this study: 1. Male participants aged 4 to 18 years; 2. Confirmed diagnosis of Duchenne muscular dystrophy (DMD) by genetic testing, with a defined genetic variant; 3. Vamorolone treatment group: participants with a regular history of prednisone use, who have switched to or plan to switch to vamorolone treatment, and who agree to comply with the required follow-up schedule; 4. Prednisone control group: participants with no prior vamorolone exposure, who have received regular prednisone treatment for >=6 months, and who agree to comply with the required follow-up schedule; 5. Written informed consent obtained from the legal guardian(s) of the participant.
Exclusion criteria
Exclusion criteria: Participants meeting **any** of the following criteria will be excluded from this study: 1. Presence of other inherited muscular diseases or neurological disorders (e.g., spinal muscular atrophy, cerebral palsy, etc.); 2. Severe failure of major organs including the heart, liver, kidney, or lung; 3. Presence of other serious conditions such as malignant tumors, severe infections, or coagulation disorders; 4. History of allergy to vamorolone, prednisone, or other study-related test reagents; 5. Current participation in other clinical trials; 6. Inability of the legal guardian to comply with follow-up requirements or refusal to provide written informed consent.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| North Star Ambulatory Assessment, NSAA;Timing Function Test; | — |
Secondary
| Measure | Time frame |
|---|---|
| Serum creatine kinase;Height Z-score;Weight Z-score;BMI Z-score;Bone mineral density;bone turnover biomarkers;bone turnover biomarkers; | — |
Countries
China
Contacts
Public ContactXiaotang Cai
West China Second University Hospital, Sichuan University
Outcome results
None listed