Early Phase 1 Muscular Dystrophy Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- The child's parent or legal guardian fully understands what the study involves and agrees to participate by signing a consent form
- The child is a boy who can walk on his own and is at least 4 years old but not yet 8 years old
- The child has been diagnosed with Duchenne Muscular Dystrophy (DMD) confirmed by genetic testing
- The child shows at least one typical sign of DMD, such as weakness in the muscles closest to the body (like hips or shoulders), a waddling walk, unusually large-looking calf muscles, difficulty getting up from the floor (pushing up using hands on legs), or shoulder blades that stick out
- The child is able to cooperate with movement/physical tests, MRI scans, and a small muscle tissue sample (biopsy) as required by the study
Who may not be able to join:
- The child has active Hepatitis B, Hepatitis C, or HIV infections (confirm with trial site)
- The child is currently receiving treatment for Hepatitis B, Hepatitis C, or HIV
- The child's heart is not pumping blood effectively enough, or they have moderate-to-severe heart failure
- The child has serious or ongoing irregular heartbeats, or was born with a heart defect
- The child's heart-related medications or treatments have been changed within 1 month before the study begins
- The child has a significant liver condition, such as scarring of the liver, high blood pressure in the liver, an enlarged spleen, liver-related brain problems, or certain abnormal findings on liver scans or blood tests that the doctor considers medically important
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Jiwen Wang, Shanghai Children's Medical Center, affiliated to Shanghai Jiao Tong University School of Medicine
Phone: 18916613192
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Incidence of dose limiting toxicity (DLT) events; The incidence of adverse events (AEs) and serious adverse events (SAEs)
Can't join this trial?
Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.