Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular Dystrophy
Status:
Recruiting
Trial end date:
2022-12-31
Target enrollment:
Participant gender:
Summary
Importance: Currently the gold standard treatment for ambulant patients is corticosteroids.
Granulocyte colony-stimulating factor (G-CSF) has been reported to exert the proliferation of
satellite cells, the regulation of myoblast proliferation, and the differentiation and
promotion of muscle regeneration and repair.
Objectives To evaluate the safety and efficacy of G-CSF in children and adolescents with
muscular dystrophies Duchenne muscular dystrophy, Becker muscular dystrophy ,
Fascioscapulohumeral dystrophy.
Design, Setting, and Participants: Patients aged 5-15 with diagnosed muscular dystrophies
will be included in an open study. Patients wheelchair-bound and and mobile and
self-independent can participate in the study. Patients also treated with steroids can
participate in this study. Clinical examination and physiotherapeutic and laboratory tests
will be perform. G-CSF (5mcg/kg/body/d) is given subcutaneously for five consecutive days
during the 1st, 2nd, 3rd. 6th and 12th months. Manual muscle testing (Lovett test) of the
upper and lower extremities, isometric force with the hand dynamometer, and the 6MWT (six
minute walk test) are measured before and after therapy.