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

February 12, 2021 updated by: Medical University of Bialystok

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Anticipated)

27

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Wojciech Kulak, MD,PhD,Prof
  • Phone Number: +48603512723
  • Email: kneur2@wp.pl

Study Contact Backup

  • Name: Dorota Sienkiewicz, MD,PhD
  • Phone Number: +48857450601
  • Email: sdorota11@op.pl

Study Locations

  • Poland
    • Podlaskie
      • Białystok, Podlaskie, Poland, 15-2174
        • Recruiting
        • Department of Pediatric Rehabilitation
        • Contact:
          • Wojciech Kułak, MD, PhD, Prof.
          • Phone Number: +48603512723
          • Email: kneur2@wp.pl
        • Contact:
          • Dorota Sienkiewicz, MD, PhD
          • Phone Number: +48857450601
          • Email: 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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years to 15 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Granulocyte colony-stimulating factor
Granulocyte colony-stimulating factor Muscle strength Muscular dystrophy
Drug: Granulocyte colony-stimulating factor (Filgrastim)
Drug administration
Other Names:
  • Filgrastim

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Seven years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Seven years
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
Seven years
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
Seven years
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.
Seven years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Medical University of Bialystok

Investigators

  • Principal Investigator: Wojciech Kulak, MD,PhD,Prof, Medical University of Bialystok, Bialystok, Poland

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 1, 2013

Primary Completion (Anticipated)

December 31, 2021

Study Completion (Anticipated)

December 31, 2022

Study Registration Dates

First Submitted

June 23, 2016

First Submitted That Met QC Criteria

June 24, 2016

First Posted (Estimate)

June 27, 2016

Study Record Updates

Last Update Posted (Actual)

February 15, 2021

Last Update Submitted That Met QC Criteria

February 12, 2021

Last Verified

February 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • UMB 143-20899 P

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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