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

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ClinicalTrials.gov Identifier: NCT02814110
Recruitment Status : Recruiting
First Posted : June 27, 2016
Last Update Posted : September 14, 2017
Sponsor:
Information provided by (Responsible Party):
Medical University of Bialystok

June 23, 2016
June 27, 2016
September 14, 2017
March 1, 2013
December 31, 2017   (Final data collection date for primary outcome measure)
Increase in muscle strength in patients with muscular dystrophy [ Time Frame: Four years ]
Same as current
Complete list of historical versions of study NCT02814110 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular Dystrophy
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.

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.

Interventional
Phase 1
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Increase of Muscle Strength in Patients With Muscular Dystrophy
Drug: Granulocyte colony-stimulating factor (Filgrastim)
Drug administration
Other Name: Filgrastim
Granulocyte colony-stimulating factor
Granulocyte colony-stimulating factor Muscle strength Muscular dystrophy
Intervention: Drug: Granulocyte colony-stimulating factor (Filgrastim)
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
27
December 31, 2018
December 31, 2017   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
5 Years to 15 Years   (Child)
No
Contact: Wojciech Kulak, MD,PhD,Prof +48603512723 kneur2@wp.pl
Contact: Dorota Sienkiewicz, MD,PhD +48857450601 sdorota11@op.pl
Poland
 
 
NCT02814110
UMB 143-20899 P
Yes
Not Provided
Plan to Share IPD: Undecided
Medical University of Bialystok
Medical University of Bialystok
Not Provided
Principal Investigator: Wojciech Kulak, MD,PhD,Prof Medical University of Bialystok, Bialystok, Poland
Medical University of Bialystok
September 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP