Phase 2 Muscular Dystrophy Trial, Recruiting

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• The participant (and/or their legal guardian) must be willing and able to give written consent after fully understanding the study's purpose, methods, and potential risks.
• Boys who are able to walk and are aged 4 years old up to (but not including) 9 years old.
• A confirmed mutation in the DMD gene, shown by genetic testing.
• Blood test results for a muscle enzyme called creatine kinase (CK) that fall within the range required by the study.
• Currently taking a stable, standard dose of steroid medication (glucocorticoids) prior to the screening period.
• Antibody levels related to the treatment's delivery system (AAV capsid antibodies) that meet the study's requirements (confirm with trial site).
• Able to participate in movement and physical function tests, MRI scans, and a small muscle tissue sample (biopsy) as required by the study.
• Blood and laboratory test results during the screening and baseline period that meet the study's required standards.
• The participant and/or their legal guardian must be willing to follow the study schedule closely and attend all required visits.

Who may not be able to join:

• People who test positive for hepatitis B, hepatitis C, HIV, or syphilis, or who have certain elevated hepatitis B virus levels in their blood.
• People currently receiving antiviral treatment for hepatitis B, hepatitis C, HIV, or similar infections.
• People whom the trial doctor considers to have severe behavioural or thinking difficulties that would make participation in the study too difficult.
• People with poorly controlled asthma, significantly reduced lung function due to Duchenne Muscular Dystrophy, or repeated episodes of infectious pneumonia that the doctor considers may affect breathing.
• People with a weakened heart pump function (left ventricular ejection fraction below 50%) or who have been assessed as having serious heart failure symptoms (confirm with trial site).
• People with serious or ongoing irregular heart rhythms, or certain types of heart conditions that the trial doctor considers unsuitable for the study.
• People whose heart-related medications were started, changed, stopped, or restarted within one month before receiving the study treatment.
• People with a history of serious liver conditions such as liver scarring (stage 3 or above), high blood pressure in the liver, or certain liver abnormalities found during screening tests.
• People who have had a serious infection such as pneumonia, kidney infection, or meningitis within 4 weeks before starting treatment (note: in some cases, joining may be able to be postponed rather than ruled out entirely — confirm with trial site).
• People who have previously received gene therapy or cell therapy, such as stem cell transplantation.
• People with a history of or current autoimmune disease, serious kidney, digestive, neurological, or blood clotting disorders, cancer, or other significant health conditions.
• People with any other condition that the trial doctor considers unsuitable for participation.

Important: Always verify eligibility with the trial site directly before applying.