Clinical trial NCT02814110: Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular Dystrophy
Official title: Efficacy and the Safety of Granulocyte Colony-stimulating Factor Treatment in Children and Adolescents With Muscular Dystrophy: An Open Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Increase Muscle Strength in Patients With Muscular Dystrophy
Keywords: Granulocyte colony-stimulating factor
muscular dystrophy
muscle strength
children
adolescents
Lead sponsor: Medical University of Bialystok
Sponsor class: OTHER
First posted: 2016-06-27
Enrollment: 27
Sex: ALL
Minimum age (years): 5
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Safety based on number of participants with adverse events.
timeFrame: Seven years
Secondary outcomes:
measure: Muscle strength in patients with muscular dystrophy
timeFrame: Seven years
measure: Laboratory investigations in patients with muscular dystrophy
timeFrame: Seven years
measure: Abdominal ultrasonography in patients with muscular dystrophy
timeFrame: Seven years
measure: Electrocardiographic records in patients with muscular dystrophy
timeFrame: Seven years
Other outcomes:
Locations:
city: Białystok
country: Poland
facility: Department of Pediatric Rehabilitation
state: Podlaskie
Study officials:
affiliation: Medical University of Bialystok, Bialystok, Poland
name: Wojciech Kulak, MD,PhD,Prof
role: PRINCIPAL_INVESTIGATOR
References:
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: 10.1155/2019/4789101. eCollection 2019.
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-Randomized Clinical Trial. Front Neurol. 2017 Oct 26;8:566. doi: 10.3389/fneur.2017.00566. eCollection 2017.
citation: 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 Monocyte Subsets in Pediatric Patients with Muscular Dystrophies. Stem Cells Int. 2016;2016:2650849. doi: 10.1155/2016/2650849. Epub 2015 Dec 6.