Hemophilia A, SevereNCT04754997
Effect of Exercise in Pediatric Hemophilia
A study testing Traditional exercise training and Specific exercise training in people with Hemophilia A, Severe and Hemophilia Arthropathy, run by Hasan Kalyoncu University at 1 site in Turkey (Türkiye).
Trial summary
This study investigates the effect of exercise training on pain, physical activity and quality of life in pediatric hemophilia patients. Read more about this study
- Who is it for?
- Ages 8 to 18 years, men only
- Conditions the study is for
- Hemophilia A, Severe, Hemophilia Arthropathy
- What is being tested?
- Traditional exercise training and Specific exercise training
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 16 Jan 2021to 30 Nov 2021
- Phase
- Interventional
- 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 8 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosed with hemophilia (Hemophilia A) by a hematologist
- Hemophilia is moderate or severe, without spontaneous bleeding complaints
- Having a history of bleeding in the knee joint
- Not doing any regular exercise
- Willing to continue working and will not start other treatment during the study
- Patients whose parents were informed about the study and whose consent form was obtained
Who cannot join
- Those who have undergone another surgery (joint debridement, fracture, knee replacement) that will affect lower extremity function
- Those with neurological disease or any sensory loss or peripheral nerve injury that may affect functional performance and balance
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site
Hasan Kalyoncu University
Gaziantep, Şahinbey, 27100
About this study
Official title: The Effect of Exercise Training on Pain, Physical Activity and Quality of Life in Pediatric Hemophilia Patients
This study investigates the effect of exercise training on pain, physical activity and quality of life in pediatric hemophilia patients.
Read the detailed description
Hemophilia is a congenital X-linked recessive disease characterized by the lack or absence of clotting factors in the blood. Male individuals with problems in the X chromosome are affected, females are carriers. There are two common types, factor VIII (Hemophilia A) and factor IX (Hemophilia B), and the percentage of the exposed factor in circulation determines the degree of hemophilia disease (<1% severe, 1-5% moderate,> 20% mild). The most common clinical symptoms in hemophilia are intra-articular and intramuscular spontaneous bleeding, a history of bleeding that lasts longer than expected after trauma, hemarthrosis and hematomas after crawling or walking in children with moderate or severe hemophilia. Pain, swelling, redness, temperature increase and limitation of movement occur in the bleeding joint. Bleeding into the joint space causes inflammation in the synovial membrane and synovitis occurs. With chronic synovitis, the synovial membrane thickens. Degeneration of the joint cartilage and narrowing of the joint space occurs. Hemarthroses constitute 70-80% of bleeding findings. The most common bleeding joints are knee joints with a rate of 45%. Besides pharmacological treatment for joint and muscle bleeding, the most suitable option is exercise therapy.
The aim of our study is to examine the effect of exercise agitation on pain, physical activity and quality of life in pediatric hemophilia patients. The results of the study indicate that the exercise protocol developed for the lower extremity in pediatric hemophilia patients will have a positive effect on pain, physical activity and quality of life.
- Conditions
- Hemophilia A, Severe, Hemophilia Arthropathy
- Being tested
- Traditional exercise training, Specific exercise training
- Keywords
- Hemophilia, Factor VIII, Closed chain exercise, Knee
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Traditional exercise trainingOther
The therapy program will planned for 12 weeks and the sessions will be performed in 45 minutes (min) for 2 days a week. The lower extremity range of motion exercise and strengthening exercises will be applied on the mat 10 repetition. The number of repetitions and type of exercises will be changed every 3 weeks.
Specific exercise trainingOther
The therapy program will planned for 12 weeks and the sessions will be performed in 45 minutes (min) for 2 days a week. Exercise training consists of lower extremity closed kinetic chain exercises and core exercises. The type of exercise changes every 3 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Control group | No intervention | This group will not take any exercise intervention. |
| Traditional exercise training group | Active comparator | This group will get traditional therapy that is include range of motion exercises and resistance exercise training. Receives: Other: Traditional exercise training |
| Specific exercise training group | Experimental | This group will get a specific programme that combinated with closed kinetic chain exercises and core exercises training Receives: Other: Specific exercise training |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Pain
Mc Gill Short Form will used to determine the type and severity of the pain. A short form of the McGill Pain Questionnaire (SF-MPQ) has been developed. The main component of the SF-MPQ consists of 15 descriptors (11 sensory; 4 affective) which are rated on an intensity scale as 0 = none, 1 = mild, 2 = moderate or 3 = severe.
Measured: through of the study, average 12 weeks
Functional ambulatory mobility
Time Up and Go test will used to determine ambulatory mobility. Time up and go test required documenting the time in seconds that subjects required to: "rise from a standard arm chair, walk to a line on the floor 3 meters away, turn, return, and sit down again." If relapsed time score is increased, that is mean mobility function will worse.
Measured: through of the study, average 12 weeks
Functional exercise capacity
Two minutes walk test will used to determine physical activity. 2-minute walk test (2MWT) is classified to represent the most suitable method to assess the submaximal level of functional exercise capacity in children. This test measures the distance that a patient or participant quickly walk on a flat, hard surface in a period of 2 minutes.If the covered distance is increased, that is mean functional capacity will worse.
Measured: through of the study, average 12 weeks
Lower extremity functional strength test
Sit to stand test will used to determine physical activity. The sit-to-stand test measures time needed to complete three consecutive sit-to-stand cycles as quickly as possible. The higher the rate of three-repetition sit-to-stand (repetitions per second), the more strength a children has.
Measured: through of the study, average 12 weeks
The quality of Life
The Children's Quality of Life Scale ( PedsQL) will used to determine to quality of life level. The quality of life scale for children questions the last month of children and adolescents. It is a 5-point Likert-type scale with never 100 points, rarely 75 points, sometimes 50 points, often 25 points and always 0 points. The total score is obtained by dividing the scores obtained from the scale by the number of filled items. The higher the total score, the better the quality of life.
Measured: through of the study, average 12 weeks
Secondary outcomes (4)
Knee Proprioception
Electro goniometer measurement will used to determine knee proprioception
Measured: through of the study, average 12 weeks
Joint Health
Hemophilia Joint Health Scale will used to determine bilaterally knee, elbow and ankle joint health. Total score of the scale is 124 point. The higher score of the scale, the more deficit joints have.
Measured: through of the study, average 12 weeks
Functional Independence
Functional Independence Score in Hemophilia (FISH) will used to functional independence. FISH is based on observing the performance of daily life activities. Patients are assessed for their ability to perform eight tasks, divided into three categories: self-care (eating, grooming, bathing and dressing), transfers (chair and squatting) and mobility (walking, going up stairs and running).The scores achieved in each task are summed giving a total from 8 to 32 points with 32 indicating the highest level of functional independence
Measured: through of the study, average 12 weeks
Static Balance
Stand on one leg test was used assess static balance
Measured: through of the study, average 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Feb 2021
- Study start16 Jan 2021
- Primary completion10 Nov 2021 (estimated)
- Study completion30 Nov 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Hemophilia A, SevereStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Participants were divided in three groups. Two groups will get two different intervention. One group will be the control group.
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hasan Kalyoncu University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Tuğba GÖNEN, Hasan Kalyoncu University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04754997
- Sponsor's protocol ID
- 2020/116
- Status last verified
- Feb 2021
- First submitted
- 8 Feb 2021
- Last updated
- 15 Feb 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
van Vulpen LFD, Holstein K, Martinoli C. Joint disease in haemophilia: Pathophysiology, pain and imaging. Haemophilia. 2018 May;24 Suppl 6:44-49. doi: 10.1111/hae.13449.
PubMed 29878659Wagner B, Kruger S, Hilberg T, Ay C, Hasenoehrl T, Huber DF, Crevenna R. The effect of resistance exercise on strength and safety outcome for people with haemophilia: A systematic review. Haemophilia. 2020 Mar;26(2):200-215. doi: 10.1111/hae.13938. Epub 2020 Feb 24.
PubMed 32091659Siqueira TC, Dominski FH, Andrade A. Effects of exercise in people with haemophilia: An umbrella review of systematic reviews and meta-analyses. Haemophilia. 2019 Nov;25(6):928-937. doi: 10.1111/hae.13868. Epub 2019 Oct 30.
PubMed 31667968Souza JC, Simoes HG, Campbell CS, Pontes FL, Boullosa DA, Prestes J. Haemophilia and exercise. Int J Sports Med. 2012 Feb;33(2):83-8. doi: 10.1055/s-0031-1286292. Epub 2011 Nov 17.
PubMed 22095329
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
- The Effect of Exercise Training on Pain, Physical Activity and Quality of Life in Pediatric Hemophilia Patients
- ClinicalTrials.gov ID
- NCT04754997
- Acronym / sponsor ID
- 2020/116
- Conditions
- Hemophilia A, Severe, Hemophilia Arthropathy
- Keywords
- Hemophilia, Factor VIII, Closed chain exercise, Knee
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Hasan Kalyoncu UniversityUniversity, hospital or other
- Study start
- 16 Jan 2021 (actual)
- Primary completion
- 10 Nov 2021 (estimated)
- Study completion
- 30 Nov 2021 (estimated)
- First posted
- 15 Feb 2021 (actual)
- 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.
This study investigates the effect of exercise training on pain, physical activity and quality of life in pediatric hemophilia patients.
Detailed description
Hemophilia is a congenital X-linked recessive disease characterized by the lack or absence of clotting factors in the blood. Male individuals with problems in the X chromosome are affected, females are carriers. There are two common types, factor VIII (Hemophilia A) and factor IX (Hemophilia B), and the percentage of the exposed factor in circulation determines the degree of hemophilia disease (<1% severe, 1-5% moderate,> 20% mild). The most common clinical symptoms in hemophilia are intra-articular and intramuscular spontaneous bleeding, a history of bleeding that lasts longer than expected after trauma, hemarthrosis and hematomas after crawling or walking in children with moderate or severe hemophilia. Pain, swelling, redness, temperature increase and limitation of movement occur in the bleeding joint. Bleeding into the joint space causes inflammation in the synovial membrane and synovitis occurs. With chronic synovitis, the synovial membrane thickens. Degeneration of the joint cartilage and narrowing of the joint space occurs. Hemarthroses constitute 70-80% of bleeding findings. The most common bleeding joints are knee joints with a rate of 45%. Besides pharmacological treatment for joint and muscle bleeding, the most suitable option is exercise therapy.
The aim of our study is to examine the effect of exercise agitation on pain, physical activity and quality of life in pediatric hemophilia patients. The results of the study indicate that the exercise protocol developed for the lower extremity in pediatric hemophilia patients will have a positive effect on pain, physical activity and quality of life.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 3
- Model description
- Participants were divided in three groups. Two groups will get two different intervention. One group will be the control group.
| Arm | Type | Receives |
|---|---|---|
| Control group | No intervention | –This group will not take any exercise intervention. |
| Traditional exercise training group | Active comparator | Other: Traditional exercise trainingThis group will get traditional therapy that is include range of motion exercises and resistance exercise training. |
| Specific exercise training group | Experimental | Other: Specific exercise trainingThis group will get a specific programme that combinated with closed kinetic chain exercises and core exercises training |
Who may qualify
- Ages
- 8 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Diagnosed with hemophilia (Hemophilia A) by a hematologist
- Hemophilia is moderate or severe, without spontaneous bleeding complaints
- Having a history of bleeding in the knee joint
- Not doing any regular exercise
- Willing to continue working and will not start other treatment during the study
Key exclusion criteria (2 listed)
- Those who have undergone another surgery (joint debridement, fracture, knee replacement) that will affect lower extremity function
- Those with neurological disease or any sensory loss or peripheral nerve injury that may affect functional performance and balance
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 |
|---|---|---|
| Traditional exercise trainingThe therapy program will planned for 12 weeks and the sessions will be performed in 45 minutes (min) for 2 days a week. The lower extremity range of motion exercise and strengthening exercises will be applied on the mat 10 repetition. The number of repetitions and type of exercises will be changed every 3 weeks. | Other | Traditional exercise training group |
| Specific exercise trainingThe therapy program will planned for 12 weeks and the sessions will be performed in 45 minutes (min) for 2 days a week. Exercise training consists of lower extremity closed kinetic chain exercises and core exercises. The type of exercise changes every 3 weeks. | Other | Specific exercise training group |
Primary outcome
The Pain
Time frame: through of the study, average 12 weeks
Mc Gill Short Form will used to determine the type and severity of the pain. A short form of the McGill Pain Questionnaire (SF-MPQ) has been developed. The main component of the SF-MPQ consists of 15 descriptors (11 sensory; 4 affective) which are rated on an intensity scale as 0 = none, 1 = mild, 2 = moderate or 3 = severe.
Functional ambulatory mobility
Time frame: through of the study, average 12 weeks
Time Up and Go test will used to determine ambulatory mobility. Time up and go test required documenting the time in seconds that subjects required to: "rise from a standard arm chair, walk to a line on the floor 3 meters away, turn, return, and sit down again." If relapsed time score is increased, that is mean mobility function will worse.
Functional exercise capacity
Time frame: through of the study, average 12 weeks
Two minutes walk test will used to determine physical activity. 2-minute walk test (2MWT) is classified to represent the most suitable method to assess the submaximal level of functional exercise capacity in children. This test measures the distance that a patient or participant quickly walk on a flat, hard surface in a period of 2 minutes.If the covered distance is increased, that is mean functional capacity will worse.
Lower extremity functional strength test
Time frame: through of the study, average 12 weeks
Sit to stand test will used to determine physical activity. The sit-to-stand test measures time needed to complete three consecutive sit-to-stand cycles as quickly as possible. The higher the rate of three-repetition sit-to-stand (repetitions per second), the more strength a children has.
The quality of Life
Time frame: through of the study, average 12 weeks
The Children's Quality of Life Scale ( PedsQL) will used to determine to quality of life level. The quality of life scale for children questions the last month of children and adolescents. It is a 5-point Likert-type scale with never 100 points, rarely 75 points, sometimes 50 points, often 25 points and always 0 points. The total score is obtained by dividing the scores obtained from the scale by the number of filled items. The higher the total score, the better the quality of life.
4 secondary outcomes
Knee Proprioception
Time frame: through of the study, average 12 weeks
Joint Health
Time frame: through of the study, average 12 weeks
Functional Independence
Time frame: through of the study, average 12 weeks
Static Balance
Time frame: through of the study, average 12 weeks
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.
- Background: PubMed 29878659van Vulpen LFD, Holstein K, Martinoli C. Joint disease in haemophilia: Pathophysiology, pain and imaging. Haemophilia. 2018 May;24 Suppl 6:44-49. doi: 10.1111/hae.13449.
- Background: PubMed 32091659Wagner B, Kruger S, Hilberg T, Ay C, Hasenoehrl T, Huber DF, Crevenna R. The effect of resistance exercise on strength and safety outcome for people with haemophilia: A systematic review. Haemophilia. 2020 Mar;26(2):200-215. doi: 10.1111/hae.13938. Epub 2020 Feb 24.
- Background: PubMed 31667968Siqueira TC, Dominski FH, Andrade A. Effects of exercise in people with haemophilia: An umbrella review of systematic reviews and meta-analyses. Haemophilia. 2019 Nov;25(6):928-937. doi: 10.1111/hae.13868. Epub 2019 Oct 30.
- Background: PubMed 22095329Souza JC, Simoes HG, Campbell CS, Pontes FL, Boullosa DA, Prestes J. Haemophilia and exercise. Int J Sports Med. 2012 Feb;33(2):83-8. doi: 10.1055/s-0031-1286292. Epub 2011 Nov 17.
- Individual participant dataSharing planned: No
Related trials for Hemophilia A, Severe
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
RecruitingPhase 3
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Hemophilia A, Hemophilia B
Active, not recruitingPhase 4
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
Severe Hemophilia A, Moderate Hemophilia A
Not yet recruitingObservational study
Post Approval Phase 4 Study to Learn More About How Safe Octocog Alfa is, and How Well it Works in Patients With Severe Hemophilia A in India
Severe Hemophilia A
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Traditional exercise training and Specific exercise training in people with Hemophilia A, Severe and Hemophilia Arthropathy, run by Hasan Kalyoncu University at 1 site in Turkey (Türkiye).
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 6 inclusion and 2 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 Turkey (Türkiye). 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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Pain (time frame: through of the study, average 12 weeks). The study lists 5 primary outcomes in all. 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 NCT04754997, which also lists 4 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.