Increase Muscle Strength in Patients With Muscular Dystrophy
Conditions
Keywords
Granulocyte colony-stimulating factor, muscular dystrophy, muscle strength, children, adolescents
Brief 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.
Detailed 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.
Interventions
Drug administration
Sponsors
Medical University of Bialystok
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Years to 15 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety based on number of participants with adverse events. | Seven years | Adverse events will be monitored and scored for severity and related to the Granulocyte Colony-Stimulating Factor administration. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Muscle strength in patients with muscular dystrophy | Seven years | The assessment of muscle strength of the upper and lower limbs by Lovett test. The evaluation isometric force of upper limbs with the hand dynamometer. Distance measurement in a 6-minute walk test (6MWT) by feet or wheelchair,6MWT before and after therapy. |
| Laboratory investigations in patients with muscular dystrophy | Seven years | Blood count: Red blood cells 10\^6/µL; Hemoglobin g/dl, Leucocytes 10\^3/µL, Platelets 10\^3/µL; biochemistry CRP - C Reactive Protein - mg/L; creatinine - mg/dL; glucose mg/dL, electrolytes, AST- U/L; ALT - U/L; cholesterol mg/dL; fibrinogen - mg/dL, partial thromboplastin time - sec; prothrombin time - sec, creatine kinase - U/L |
| Abdominal ultrasonography in patients with muscular dystrophy | Seven years | Abdominal ultrasonography with a spleen measurement will be done before and after G-CSF administration |
| Electrocardiographic records in patients with muscular dystrophy | Seven years | Electrocardiographic records wiil ibe done in patients with muscular Dystrophy before and after G-CSF administration. |
Countries
Poland
Contacts
Primary ContactWojciech Kulak, MD,PhD,Prof
Backup ContactDorota Sienkiewicz, MD,PhD
Outcome results
None listed