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

Medical University of Bialystok

Status and phase: Unknown; Phase 1

Conditions: Increase Muscle Strength in Patients With Muscular Dystrophy

Treatments: Drug: Granulocyte colony-stimulating factor (Filgrastim)

Study type: Interventional

Funder types: Other

Identifiers

NCT02814110

UMB 143-20899 P

Details and patient eligibility

About

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.

Full description

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. Blood is sampled before G-CSF administration and on the 5th day of each treatment cycle.

During each cycle of G-CSF administration physical therapy is also applied. Abdominal ultrasonography with a spleen assessment is performed before and after 7 days of G-CSF administration. Spirometry and electrocardiographic record are also performed. Side effects of G-CSF treatment will be assessed.

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. MRI of the gastrocnemius muscles will performed at the beginning and at the end of therapy.

Enrollment: 27 estimated patients

Sex: All

Ages: 5 to 15 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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27 participants in 1 patient group

Granulocyte colony-stimulating factor

Other group

Description:

Granulocyte colony-stimulating factor Muscle strength Muscular dystrophy

Treatment:

Drug: Granulocyte colony-stimulating factor (Filgrastim)