Increase Muscle Strength in Patients With Muscular Dystrophy Clinical Trial
Official title:
Efficacy and the Safety of Granulocyte Colony-stimulating Factor Treatment in Children and Adolescents With Muscular Dystrophy: An Open Study
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.
| Status | Recruiting |
| Enrollment | 27 |
| Est. completion date | December 31, 2022 |
| Est. primary completion date | December 31, 2021 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 5 Years to 15 Years |
| Eligibility | 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 |
| Country | Name | City | State |
|---|---|---|---|
| Poland | Department of Pediatric Rehabilitation | Bialystok | Podlaskie |
| Lead Sponsor | Collaborator |
|---|---|
| Medical University of Bialystok |
Poland,
Eljaszewicz A, Sienkiewicz D, Grubczak K, Okurowska-Zawada B, Paszko-Patej G, Miklasz P, Singh P, Radzikowska U, Kulak W, Moniuszko M. Effect of Periodic Granulocyte Colony-Stimulating Factor Administration on Endothelial Progenitor Cells and Different Mo — View Citation
Sienkiewicz D, Kulak W, Okurowska-Zawada B, Paszko-Patej G, Wojtkowski J, Sochon K, Kalinowska A, Okulczyk K, Sienkiewicz J, McEachern E. Efficacy and the Safety of Granulocyte Colony-Stimulating Factor Treatment in Patients with Muscular Dystrophy: A Non — View Citation
Sienkiewicz D, Kulak W, Paszko-Patej G, Okurowska-Zawada B, Sienkiewicz J, Kulak P. Biochemical Changes in Blood of Patients with Duchenne Muscular Dystrophy Treated with Granulocyte-Colony Stimulating Factor. Biomed Res Int. 2019 Mar 13;2019:4789101. doi — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Safety based on number of participants with adverse events. | Adverse events will be monitored and scored for severity and related to the Granulocyte Colony-Stimulating Factor administration. | Seven years | |
| Secondary | Muscle strength in patients with muscular dystrophy | 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. |
Seven years | |
| Secondary | Laboratory investigations in patients with muscular dystrophy | 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 | Seven years | |
| Secondary | Abdominal ultrasonography in patients with muscular dystrophy | Abdominal ultrasonography with a spleen measurement will be done before and after G-CSF administration | Seven years | |
| Secondary | Electrocardiographic records in patients with muscular dystrophy | Electrocardiographic records wiil ibe done in patients with muscular Dystrophy before and after G-CSF administration. | Seven years |