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Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients

key information

study id #: NCT02196467

condition: Duchenne Muscular Dystrophy

status: recruiting

purpose:

This Phase I/II of the clinical trial is to investigate whether the transplantation of normal myoblasts throughout one muscle (the extensor carpi radialis) of the patients is safe and will improve the strength of that muscle. During this Phase I/II, the patients will be transplanted with myoblasts grown from the muscle biopsy of a donor and kept frozen in liquid nitrogen. Thirty million myoblasts will be injected per cm cube in a progressively higher surface of the radialis (i.e., 3, 6 and 9 cm2). The contralateral muscle will be injected with saline to serve as a control. The strength of both muscles will be measured at 3 months post transplantation to verify whether the myoblast transplantation improved the strength of the muscle. If there is no significant strength improvement, the protocol will be terminated immediately for that patient. If there is a significant strength improvement, the patient will be maintained under immunosuppression until 6 months post transplant and his strength will be re-evaluated.

intervention: Myoblast transplantation, Saline injection

mechanism of action: Stem cell therapy to promote cellular regeneration

results: https://clinicaltrials.gov/ct2/show/results/NCT02196467

last updated: December 04, 2019