Increase Muscle Strength in Patients With Muscular DystrophyPhase 1NCT02814110
Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular Dystrophy
A Phase 1 study testing Granulocyte colony-stimulating factor (Filgrastim) in people with Increase Muscle Strength in Patients With Muscular Dystrophy, run by Medical University of Bialystok at 1 site in Poland.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 15 years
- Conditions the study is for
- Increase Muscle Strength in Patients With Muscular Dystrophy
- What is being tested?
- Granulocyte colony-stimulating factor (Filgrastim)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 sitePoland
- Study dates
- 1 Mar 2013to 31 Dec 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 15 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Muscular dystrophy - Duchenne muscular dystrophy, Becker muscular dystrophy, Fascioscapulohumeral dystrophy
- age 5-15
Who cannot join
- non- muscular dystrophy
- age below 5 years
- age over 15 years
Trial locations and contacts
1 site in 1 country.
Poland1 site, 1 recruiting
- Recruiting
Department of Pediatric Rehabilitation
Bialystok, Podlaskie Voivodeship, 15-2174
Wojciech Kułak, MD, PhD, Prof., +48603512723, kneur2@wp.pl
Dorota Sienkiewicz, MD, PhD, +48857450601, sdorota11@op.pl
About this study
Official title: 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.
Read the 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.
- Being tested
- Granulocyte colony-stimulating factor (Filgrastim)
- Keywords
- Granulocyte colony-stimulating factor, muscular dystrophy, muscle strength, children, adolescents
- Medical subject headings
- Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Granulocyte colony-stimulating factor (Filgrastim)Drug
Also called Filgrastim
Drug administration
| Study group | Role | What they receive |
|---|---|---|
| Granulocyte colony-stimulating factor | Other | Granulocyte colony-stimulating factor Muscle strength Muscular dystrophy Receives: Drug: Granulocyte colony-stimulating factor (Filgrastim) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Seven years
Secondary outcomes (4)
Muscle strength in patients with muscular dystrophy
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.
Measured: Seven years
Laboratory investigations in patients with muscular dystrophy
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
Measured: Seven years
Abdominal ultrasonography in patients with muscular dystrophy
Abdominal ultrasonography with a spleen measurement will be done before and after G-CSF administration
Measured: Seven years
Electrocardiographic records in patients with muscular dystrophy
Electrocardiographic records wiil ibe done in patients with muscular Dystrophy before and after G-CSF administration.
Measured: Seven years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2016 (estimated)
- Study start1 Mar 2013
- Primary completion31 Dec 2021 (estimated)
- Study completion31 Dec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Increase Muscle Strength in Patients With Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Medical University of Bialystok
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Wojciech Kulak, MD,PhD,Prof, Principal investigator, Medical University of Bialystok, Bialystok, Poland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02814110
- Sponsor's protocol ID
- UMB 143-20899 P
- Status last verified
- Feb 2021
- First submitted
- 23 Jun 2016
- Last updated
- 15 Feb 2021
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Publications
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.
PubMed 31001554Sienkiewicz 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.
PubMed 29123500Eljaszewicz 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.
PubMed 26770204
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and the Safety of Granulocyte Colony-stimulating Factor Treatment in Children and Adolescents With Muscular Dystrophy: An Open Study
- ClinicalTrials.gov ID
- NCT02814110
- Acronym / sponsor ID
- UMB 143-20899 P
- Keywords
- Granulocyte colony-stimulating factor, muscular dystrophy, muscle strength, children, adolescents
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 27 participants (estimated)
- Lead sponsor
- Medical University of BialystokUniversity, hospital or other
- Study start
- 1 Mar 2013 (actual)
- Primary completion
- 31 Dec 2021 (estimated)
- Study completion
- 31 Dec 2022 (estimated)
- First posted
- 27 Jun 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Granulocyte colony-stimulating factor | Other | Drug: Granulocyte colony-stimulating factor (Filgrastim)Granulocyte colony-stimulating factor Muscle strength Muscular dystrophy |
Who may qualify
- Ages
- 5 to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Muscular dystrophy - Duchenne muscular dystrophy, Becker muscular dystrophy, Fascioscapulohumeral dystrophy
- age 5-15
Key exclusion criteria (3 listed)
- non- muscular dystrophy
- age below 5 years
- age over 15 years
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Granulocyte colony-stimulating factor (Filgrastim)Also: FilgrastimDrug administration | Drug | Granulocyte colony-stimulating factor |
Primary outcome
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.
4 secondary outcomes
Muscle strength in patients with muscular dystrophy
Time frame: Seven years
Laboratory investigations in patients with muscular dystrophy
Time frame: Seven years
Abdominal ultrasonography in patients with muscular dystrophy
Time frame: Seven years
Electrocardiographic records in patients with muscular dystrophy
Time frame: Seven years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Feb 2021.
- Results publication: PubMed 31001554Sienkiewicz 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.
- Results publication: PubMed 29123500Sienkiewicz 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.
- Results publication: PubMed 26770204Eljaszewicz 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.
- Individual participant dataSharing planned: Undecided
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wojciech Kulak, MD,PhD,Prof principal investigator
Medical University of Bialystok, Bialystok, Poland
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Granulocyte colony-stimulating factor (Filgrastim) in people with Increase Muscle Strength in Patients With Muscular Dystrophy, run by Medical University of Bialystok at 1 site in Poland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 15 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Wojciech Kulak, MD,PhD,Prof (+48603512723, kneur2@wp.pl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety based on number of participants with adverse events. (time frame: Seven years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02814110, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Feb 2021.