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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 : February 15, 2021
Sponsor:
Information provided by (Responsible Party):
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.


Condition or disease Intervention/treatment Phase
Increase Muscle Strength in Patients With Muscular Dystrophy Drug: Granulocyte colony-stimulating factor (Filgrastim) Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 27 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy and the Safety of Granulocyte Colony-stimulating Factor Treatment in Children and Adolescents With Muscular Dystrophy: An Open Study
Actual Study Start Date : March 1, 2013
Estimated Primary Completion Date : December 31, 2021
Estimated Study Completion Date : December 31, 2022


Arm Intervention/treatment
Granulocyte colony-stimulating factor
Granulocyte colony-stimulating factor Muscle strength Muscular dystrophy
Drug: Granulocyte colony-stimulating factor (Filgrastim)
Drug administration
Other Name: Filgrastim




Primary Outcome Measures :
  1. Safety based on number of participants with adverse events. [ Time Frame: Seven years ]
    Adverse events will be monitored and scored for severity and related to the Granulocyte Colony-Stimulating Factor administration.


Secondary Outcome Measures :
  1. Muscle strength in patients with muscular dystrophy [ Time Frame: 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.


  2. Laboratory investigations in patients with muscular dystrophy [ Time Frame: 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

  3. Abdominal ultrasonography in patients with muscular dystrophy [ Time Frame: Seven years ]
    Abdominal ultrasonography with a spleen measurement will be done before and after G-CSF administration

  4. Electrocardiographic records in patients with muscular dystrophy [ Time Frame: Seven years ]
    Electrocardiographic records wiil ibe done in patients with muscular Dystrophy before and after G-CSF administration.



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Ages Eligible for Study:   5 Years to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02814110


Contacts
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Contact: Wojciech Kulak, MD,PhD,Prof +48603512723 kneur2@wp.pl
Contact: Dorota Sienkiewicz, MD,PhD +48857450601 sdorota11@op.pl

Locations
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Poland
Department of Pediatric Rehabilitation Recruiting
Białystok, Podlaskie, Poland, 15-2174
Contact: Wojciech Kułak, MD, PhD, Prof.    +48603512723    kneur2@wp.pl   
Contact: Dorota Sienkiewicz, MD, PhD    +48857450601    sdorota11@op.pl   
Principal Investigator: Wojciech Kułak, MD, PhD, Prof.         
Sub-Investigator: Dorota Sienkiewicz, MD, PhD         
Sub-Investigator: Bożena Okurowska-Zawada, MD, PhD         
Principal Investigator: Grażyna Paszko-Patej, MD, PhD         
Sub-Investigator: Janusz Wojtkowski, MSc         
Sub-Investigator: Karolina Sochoń, MSc         
Sub-Investigator: Kamila Okulczyk, MSc, PhD         
Sub-Investigator: Anna Kalinowska, MSc, PhD         
Sponsors and Collaborators
Medical University of Bialystok
Investigators
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Principal Investigator: Wojciech Kulak, MD,PhD,Prof Medical University of Bialystok, Bialystok, Poland
Publications of Results:
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Responsible Party: Medical University of Bialystok
ClinicalTrials.gov Identifier: NCT02814110    
Other Study ID Numbers: UMB 143-20899 P
First Posted: June 27, 2016    Key Record Dates
Last Update Posted: February 15, 2021
Last Verified: February 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by Medical University of Bialystok:
Granulocyte colony-stimulating factor
muscular dystrophy
muscle strength
children
adolescents
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lenograstim
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs