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

Phase 1

• Conditions: Increase Muscle Strength in Patients With Muscular Dystrophy
• Interventions: Drug: Granulocyte colony-stimulating factor (Filgrastim)

• Registration Number: NCT02814110
• Lead Sponsor: Medical University of Bialystok

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.

Study Timeline

• Study Start: 2013-03-01
• Study First Submitted: 2016-06-23
• Study First Submitted QC: 2016-06-24
• Study First Posted: 2016-06-27
• Status Verified: 2021-02
• Last Update Submitted: 2021-02-12
• Last Update Posted: 2021-02-15
• Primary Completion: 2021-12-31
• Study Completion: 2022-12-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 27

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Granulocyte colony-stimulating factor	Granulocyte colony-stimulating factor (Filgrastim)	Granulocyte colony-stimulating factor Muscle strength Muscular dystrophy

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Department of Pediatric Rehabilitation; 🇵🇱 Białystok, Podlaskie, Poland