Duchenne Muscular Dystrophy Clinical Trial
Official title:
Efficacy of Allogenic Mesenchymal Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2
Duchenne Muscular Dystrophy (DMD) is a X-linked genetic disorder primarily affecting males, resulting in an absence of dystrophin which ultimately leads to progressive muscle degeneration. Patients with DMD progressively lose functional abilities of movement, breath, and eventually the ability to circulate blood. Currently, there is no cure for DMD, although several strategies are being tested for treatment, none have yet proven to be sufficient. Children with DMD are generally divided into two groups based on severity or progression of the disease, non-ambulatory and ambulatory. Ambulatory patients are capable of walking independently while non-ambulatory patients cannot walk independently.
Duchenne Muscular Dystrophy (DMD) is a X-linked genetic disorder primarily affecting males,
resulting in an absence of dystrophin which ultimately leads to progressive muscle
degeneration. Patients with DMD progressively lose functional abilities of movement, breath,
and eventually the ability to circulate blood. Currently, there is no cure for DMD, although
several strategies are being tested for treatment, none have yet proven to be sufficient.
Children with DMD are generally divided into two groups based on severity or progression of
the disease, non-ambulatory and ambulatory. Ambulatory patients are capable of walking
independently while non-ambulatory patients cannot walk independently.
The purpose of this study is to investigate the effects of Allogenic Mesenchymal Stem Cell
Therapy in Ambulatory and Non-ambulatory Children with Duchenne Muscular Dystrophy and
determine its suitability as a form of treatment.
;
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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