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

NCT02814110 | Unknown Phase 1 DMC

• 1 other identifier: UMB 143-20899 P
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

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.

Conditions

Increase Muscle Strength in Patients With Muscular Dystrophy

Keywords

Granulocyte colony-stimulating factor; muscular dystrophy; muscle strength; children; adolescents

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Muscle strength in patients with muscular dystrophy

  Seven years

• Laboratory investigations in patients with muscular dystrophy

  Seven years

• Abdominal ultrasonography in patients with muscular dystrophy

  Seven years

• Electrocardiographic records in patients with muscular dystrophy

  Seven years

Study Arms (1)

Granulocyte colony-stimulating factor

OTHER

Granulocyte colony-stimulating factor Muscle strength Muscular dystrophy

Drug: Granulocyte colony-stimulating factor (Filgrastim)

Interventions

Granulocyte colony-stimulating factor (Filgrastim) DRUG

Drug administration

Also known as: Filgrastim

Granulocyte colony-stimulating factor

Eligibility Criteria

• Age: 5 Years - 15 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Muscular dystrophy - Duchenne muscular dystrophy, Becker muscular dystrophy, Fascioscapulohumeral dystrophy
• age 5-15

You may not qualify if:

• non- muscular dystrophy
• age below 5 years
• age over 15 years

Sponsors & Collaborators

• Medical University of Bialystok: lead

Study Sites (1)

Department of Pediatric Rehabilitation

Bialystok, Podlaskie Voivodeship, 15-2174, Poland

RECRUITING

Related Publications (3)

• 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.

  PMID: 31001554 RESULT

• 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.

  PMID: 29123500 RESULT

• 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.

  PMID: 26770204 RESULT

MeSH Terms

Conditions

Muscular Dystrophies

Interventions

Granulocyte Colony-Stimulating Factor; Filgrastim

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Wojciech Kulak, MD,PhD,Prof

  Medical University of Bialystok, Bialystok, Poland

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Wojciech Kulak, MD,PhD,Prof

CONTACT

+48603512723; kneur2@wp.pl

Dorota Sienkiewicz, MD,PhD

CONTACT

+48857450601; sdorota11@op.pl

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 23, 2016
• First Posted: June 27, 2016
• Study Start: March 1, 2013
• Primary Completion: December 31, 2021
• Study Completion: December 31, 2022
• Last Updated: February 15, 2021

Record last verified: 2021-02

Locations

PL (1)