Duchenne muscular dystrophy
Conditions
Interventions
Treatment group:Intramuscular injection of paternally derived muscle satellite cells
Sponsors
The First Affiliated Hospital of Soochow University
Eligibility
Sex/Gender
Male
Age
5 Years to 15 Years
Inclusion criteria
Inclusion criteria: 1. Patients with Duchenne muscular dystrophy confirmed by muscle pathology and genetic testing; 2. The patient's disease stage is moderate to severe and the disease has progressed in the past 6 months; 3. Father and patient meet HLA matching criteria for stem cell transplantation; 4. The patient and the father have no specific or serious comorbidities; 5. The patient is less than 15 years old; the father is not more than 45 years old and in good health; 6. The patient can communicate verbally or non-verbally; 7. Good compliance, willing to follow and ensure completion of the test process; 8. Informed consent must be signed.
Exclusion criteria
Exclusion criteria: 1. Patients with a defined psychiatric illness, or other neurodegenerative diseases or serious systemic diseases; 2. Have severe other systemic diseases or other diseases that may reduce the expectation of survival, such as diabetes (poor blood glucose control), myocardial infarction, unstable angina, liver cirrhosis, acute glomerulonephritis, etc.; 3. The patient is complicated with tumor; 4. Patients with ECG showing serious arrhythmias (such as ventricular tachycardia, frequent supraventricular tachycardia, atrial fibrillation, atrial flutter, etc.) or cardiac conduction abnormalities of degree II or above; 5. the patient has leukopenia (white blood cells less than 4 x 10^9/L) or agranulocytosis (white blood cells less than 1.5 x 10^9/L or neutrophils less than 0.5 x 10^9/L) of any cause; 6. Severe renal impairment with serum creatinine greater than 1.5 times the upper limit of normal; 7. The patient has liver disease or impaired liver function: ALT, AST, total bilirubin > 2 x ULN, or the patient has hepatitis virus infection; 8. The patient has a viral infection or history of viral infection that the investigator considers to affect the clinical trial; 9. The patient is receiving hormone, immunoglobulin, or immunosuppressant therapy for or within 6 months; 10. Patients with a history of severe infection in the last six months; 11. Patients with respiratory failure requiring supplementary ventilation; 12. Patient cannot adapt to MRI examination; 13. Subjects who have been enrolled in any other clinical trials within 3 months prior to patient enrollment; 14. Father with hepatitis virus infection, syphilis, or HIV positive antibody test; 15. The investigator determined that the father was not healthy enough to undergo a muscle biopsy; 16. The patient or father has a history of alcohol or illicit drug abuse; 17. Patients or guardians were unable to understand informed consent and study purposes; 18. There may be any conditions that the investigator considers to increase risk to the subject or interfere with the clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse reactions;Protein dystrophin;Motor function assessment;Muscle imaging assessment; | — |
Secondary
| Measure | Time frame |
|---|---|
| Creatine kinase;Electrophysiological assessment; | — |
Countries
China
Contacts
Public ContactFang Qi
The First Affiliated Hospital of Soochow University
Outcome results
None listed