Overview

Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular Dystrophy

Status:
Recruiting

Trial end date:
2022-12-31

Target enrollment:
0

Participant gender:
All

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.

Phase:

Phase 1

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Medical University of Bialystok

Treatments:

Lenograstim
Sargramostim

Criteria

Inclusion Criteria:

- Muscular dystrophy - Duchenne muscular dystrophy, Becker muscular dystrophy,
Fascioscapulohumeral dystrophy

- age 5-15

Exclusion Criteria:

- non- muscular dystrophy

- age below 5 years

- age over 15 years