Hemophilia ANCT07190118
Effects of Emicizumab Treatment in Patients With Hemophilia A
An observational study of Hemophilia A and Musculoskeletal Assessment, run by Hasan Kalyoncu University at 1 site in Turkey (Türkiye).
Trial summary
The aim of our study is to investigate the effects of emicizumab treatment on the musculoskeletal system in children with hemophilia A. Read more about this study
- Who is it for?
- Ages 1 year to 18 years, men only
- Conditions the study is for
- Hemophilia A, Musculoskeletal Assessment, Musculoskeletal Abnormalities
- How many people
- 20 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 30 Sep 2025to 10 Feb 2026
- How long will I take part?
- 6 Months
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 1 year to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Male patients under 18 years of age diagnosed with hemophilia A will constitute the study population.
Who may be able to join
- Children who are under 18 years of age,
- Have been diagnosed with Hemophilia A by a physician,
- Have a factor level of 1% (IU) or less,
- Have inhibitors positive or negative,
- Have no obstacles to starting subcutaneous (emicizumab) treatment,
- Willing to participate in the study
Who cannot join
- Children with a history of cerebrovascular bleeding who present any neurological findings that would prevent participation in the study
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Turkey (Türkiye)1 site
Hasan Kalyoncu University
Gaziantep, Gaziantep, (505) 090-5846
About this study
Official title: Effects of Emicizumab Treatment on the Musculoskeletal System in Children With Hemophilia A: A Prospective Study
The aim of our study is to investigate the effects of emicizumab treatment on the musculoskeletal system in children with hemophilia A.
Read the detailed description
Hemophilia is a rare inherited coagulation disorder resulting from a deficiency of factor (F) VIII or IX. It is a chronic group of diseases that primarily manifests as intra-articular (hemarthrosis) and intramuscular (hematoma) bleeding and affects quality of life. Easy ecchymosis formation in early childhood, especially spontaneous intra-articular and intramuscular bleeding, and a history of bleeding lasting longer than expected after interventions and trauma should raise the suspicion of hemophilia. The severity of bleeding findings is directly related to the degree of FVIII or FIX deficiency. While the clinical picture varies depending on the duration and frequency of bleeding, the severity of the disease is classified as severe (<1% IU dL-1), moderate (1%-5% IU dL-1), and mild (5%-40% IU dL-1). Hemophilia A occurs in 85% of the population (1 in 5,000 male births), while this rate is 15% in Hemophilia B (1 in 30,000 male births). Treatment of hemophilia is primarily divided into treatment of acute bleeding and prophylaxis. The primary goal in the treatment of acute bleeding is to achieve hemostasis within the first two hours after the onset of symptoms. Patients often recognize a bleeding episode by a tingling sensation or an "aura" before the bleeding begins. In such cases, factor replacement therapy should be administered immediately. In a patient presenting with severe acute bleeding, after determining the location and severity of the bleeding, factor VIII or factor IX and high-dose clotting factor concentrate (CFC) are administered. Factor concentrate doses should be 50 IU/kg factor VIII or 100-120 IU/kg factor IX. Even if bleeding slows or stops, CFC should be administered to maintain healing. Frequent measurement of factor levels should be performed to ensure that desired levels are maintained. Acetylsalicylic acid (ASA) and nonsteroidal anti-inflammatory drugs (NSAIDs) should be avoided during pain management due to their effects on platelet function and increased risk of bleeding. Prophylaxis treatment includes treating acute bleeding as well as replacing the missing factor in cases where there is no bleeding. Prophylactic treatment has many advantages. Prophylaxis is generally initiated at an early age to prevent and reduce the risk of joint bleeding and the development of hemophilic arthropathy. Dosing is personalized and adjusted according to the severity of bleeding. It protects joint health by reducing hemarthrosis episodes and reduces the need for joint surgical interventions. Prophylactic factor replacement in hemophilia treatment is administered two or three times a week via intravenous infusion. Emicizumab, one of the alternative pharmacological treatments developed in recent years, is a subcutaneous treatment method that provides patients with access to the medication within clinical and financial healthcare services in Turkey as of May 2025. Emicizumab, Fitusiran, and Concizumab, both inhibit natural anticoagulant pathways. With these subcutaneous treatments, injection frequency can vary depending on the molecule, but can be daily, weekly, or monthly. One of the most important advantages of these treatments is their effectiveness in the presence of alloantibodies, which are a significant cause of morbidity and mortality in hemophilia. Treatment features such as reduced injection frequency of factor replacements administered subcutaneously, eliminating the need for intravenous infusions, and increasing physical activity opportunities are thought to produce significant benefits affecting quality of life, in addition to controlling and preventing bleeding. Therefore, new drugs that promise less bleeding with fewer and more comfortable injections are quite attractive. It is believed that reduced bleeding will equally reduce musculoskeletal damage, support improved joint health, and increase the individual's level of functionality, resulting in improved quality of life by ensuring independence in daily life. Improved musculoskeletal health and independence in daily life will increase children's exercise habits. The positive contributions to the musculoskeletal system, such as increased overall muscle mass and maintenance of joint health, will also increase the effectiveness of physiotherapy programs. There are no other studies examining the effects of this newly introduced drug on the musculoskeletal system in children in Turkey. Therefore, the aim of our study was to investigate the effects of emicizumab treatment on the musculoskeletal system in children with hemophilia A.
- Keywords
- Hemophilia A, Emicizumab, Musculoskeletal system, Measurement
- Medical subject headings
- Hemophilia A, Musculoskeletal Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia A Group | This will be the first assessment before the child begins treatment with emicizumab. During the first month of treatment, the child and parents will be asked to note any joint or muscle bleeding, including the bleeding site, duration of bleeding, and whether hospitalization is necessary. At the end of the first month, parents will be contacted by phone to assess bleeding episodes, and any bleeding that occurred during the first month will be recorded. The same assessments will then be repeated at the third and sixth months of treatment. The effectiveness of the treatment will be assessed after the first six months through an analysis of the assessments. |
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 6 months
Hemophilia Joint Health Score (HJHS)
Hemophilia Joint H is a scoring system that targets the evaluation of three major joints (elbow, knee, and ankle). Consists of a total of 9 questions: 8 questions assessing joints and 1 question providing a global gait score. The hemophilia joint health score is calculated by assessing joint swelling, the longevity of the swelling, the presence of atrophy in the surrounding soft tissue, crepitus during active movement, loss of flexion or extension, pain, muscle strength, and the total global gait score. Hemophilia Joint Health Score values range from 0 to 124, with higher scores indicating poorer joint health.
Measured: through of the study, average 6 months
Lower extremity functionality and mobility- 5-Time Sit-to-Go Test
The 5-time sit-to-stand test is scored based on the time (in seconds to the nearest decimal place) in which a patient can move from a sitting position to a standing position and back to a sitting position five times. At the beginning of the test, the test participant is instructed to sit in a chair with their back straight. They are also instructed to fold their arms across their chest. The test participant should then be instructed to sit and stand five times as quickly as possible. The shorter the time taken to complete the test, the better the test result.
Measured: through of the study, average 6 months
Upper extremity functionality
The Questionnaire for Arm, Shoulder, and Hand Disabilities (Q-DASH) will be used to assess individuals' upper extremity functions. This questionnaire is a self-administered measurement tool with validated validity and reliability in Turkish. The questionnaire, consisting of 30 questions, assesses the individual's ability to perform functional activities (21 items), pain (5 items), and psychosocial aspects of the disease (4 items). The total score ranges from 0 to 100, with a higher score indicating better outcomes.
Measured: through of the study, average 6 months
Static balance
The One-Legged Standing Test will be administered to assess patients' static balance. In the one-leg standing test, the subject will stand with their arms hanging at their sides and lift each lower extremity one leg at a time, measuring the duration of their stance. The time will be stopped if they reposition their supporting leg, touch the ground with their raised foot, or seek support from an observer or any other support.
Measured: through of the study, average 6 months
Lower extremity functionality and mobility- 30-second Sit-to-Go Test
The 30-Second Chair Test is administered using a folding chair without arms. The chair, with rubber feet, is placed against a wall to prevent movement. The participant sits in the center of the chair, back straight, feet approximately shoulder-width apart and slightly behind the knees, with one foot slightly in front of the other to assist with balance. Arms are crossed at the wrists and held toward the chest. The participant is encouraged to perform as many complete poses as possible in 30 seconds. The participant is asked to sit fully between each pose. The tester, monitoring the participant's performance to ensure proper form, silently counts the number of each pose. The score is the total number of poses performed in the 30 seconds (more than half the poses by the end of 30 seconds are considered complete poses).
Measured: through of the study, average 6 months
Lower extremity functionality and mobility- Timed Up-and-Go Test
The Timed Up and Go Test (TUG) is a quick and easy-to-administer clinical test that assesses an individual's basic mobility skills. The test involves the patient rising from a standard chair, walking a distance of 3 meters (10 feet), turning around, returning to the chair, and sitting down again. The time taken to complete this task is recorded in seconds.
Measured: through of the study, average 6 months
Secondary outcomes (3)
Fear of movement
The Tampa Kinesiophobia Scale will be used to assess patients' fear of movement. The questionnaire, which will be used to measure individuals' perspectives on movement and their fear aspects, consists of 17 questions with total scores ranging from 17 to 68. Tampa scores greater than 37 indicate a high degree of kinesiophobia.
Measured: through of the study, average 6 months
Functional independence levels
The scoring system, categorized according to the International Classification of Functioning, Disability, and Health (ICF), was developed by considering activities that may be affected in the daily lives of hemophilia patients. Being a performance-based scale, it can be used in different languages. The scale consists of eight questions covering the subparameters of self-care, transfer, and locomotion. The self-care subparameter includes eating and hygiene, bathing and dressing; the transfer subparameter includes chair and squatting; and the locomotion subparameter includes walking, climbing and descending stairs (12-14 steps), and running. Activities are scored on a scale of 1 to 4, depending on the degree of independence. The minimum score is 8, and the maximum is 32. A high score indicates an individual's functional independence.
Measured: through of the study, average 6 months
Children's Quality of Life
The Children's Quality of Life Inventory (PedsQL) will be used to assess patients' quality of life. The scale consists of both child and parent assessment forms and assesses four subscales: physical functioning, social functioning, school life, and emotional well-being. The scale, consisting of 5-point Likert-type questions, measures the child's quality of life based on their life experiences over the past week. Scores from each subscale range from 0 to 100. Higher scores indicate better quality of life, while lower scores indicate problems in the relevant area. The parent and child forms can be evaluated separately to obtain results from different perspectives.
Measured: through of the study, average 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2025
- Study start30 Sep 2025 (estimated)
- Primary completion10 Nov 2025 (estimated)
- Study completion10 Feb 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (14)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies 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
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
- Target duration
- 6 Months
Sponsor and investigators
- Lead sponsor
- Hasan Kalyoncu University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Tuğba GÖNEN, Hasan Kalyoncu University
- Investigators
- Tuğba GÖNEN, Asisst. Prof. Dr., Principal investigator, Hasan Kalyoncu University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07190118
- Sponsor's protocol ID
- 2025/096
- Status last verified
- Sep 2025
- First submitted
- 1 Sep 2025
- Last updated
- 24 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Poonnoose PM, Manigandan C, Thomas R, Shyamkumar NK, Kavitha ML, Bhattacharji S, Srivastava A. Functional Independence Score in Haemophilia: a new performance-based instrument to measure disability. Haemophilia. 2005 Nov;11(6):598-602. doi: 10.1111/j.1365-2516.2005.01142.x.
PubMed 16236109Kumban W, Amatachaya S, Emasithi A, Siritaratiwat W. Five-times-sit-to-stand test in children with cerebral palsy: reliability and concurrent validity. NeuroRehabilitation. 2013;32(1):9-15. doi: 10.3233/NRE-130818.
PubMed 23422454McLaughlin P, Morris R, Chowdary P. Investigating the relationship between the HJHS and HAL in routine clinical practice: A retrospective review. Haemophilia. 2018 Nov;24(6):988-994. doi: 10.1111/hae.13614. Epub 2018 Oct 8.
PubMed 30295404R CB, A TO, S PA, J CJ, F Q, J NM, Ja LP. Using the Hemophilia Joint Health Score for assessment of children: Reliability of the Spanish version. Physiother Theory Pract. 2019 Apr;35(4):341-347. doi: 10.1080/09593985.2018.1443356. Epub 2018 Feb 27.
PubMed 29485331Sun J, Hilliard PE, Feldman BM, Zourikian N, Chen L, Blanchette VS, Luke KH, Poon MC. Chinese Hemophilia Joint Health Score 2.1 reliability study. Haemophilia. 2014 May;20(3):435-40. doi: 10.1111/hae.12330. Epub 2013 Dec 16.
PubMed 24330460Hilliard P, Funk S, Zourikian N, Bergstrom BM, Bradley CS, McLimont M, Manco-Johnson M, Petrini P, van den Berg M, Feldman BM. Hemophilia joint health score reliability study. Haemophilia. 2006 Sep;12(5):518-25. doi: 10.1111/j.1365-2516.2006.01312.x.
PubMed 16919083Yakut Y, Yakut E, Bayar K, Uygur F. Reliability and validity of the Turkish version short-form McGill pain questionnaire in patients with rheumatoid arthritis. Clin Rheumatol. 2007 Jul;26(7):1083-7. doi: 10.1007/s10067-006-0452-6. Epub 2006 Nov 15.
PubMed 17106618Steen Carlsson K, Andersson E, Berntorp E. Preference-based valuation of treatment attributes in haemophilia A using web survey. Haemophilia. 2017 Nov;23(6):894-903. doi: 10.1111/hae.13322. Epub 2017 Aug 29.
PubMed 28851125Meeks SL, Batsuli G. Hemophilia and inhibitors: current treatment options and potential new therapeutic approaches. Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):657-662. doi: 10.1182/asheducation-2016.1.657.
PubMed 27913543Franchini M, Mannucci PM. Non-factor replacement therapy for haemophilia: a current update. Blood Transfus. 2018 Sep;16(5):457-461. doi: 10.2450/2018.0272-17. Epub 2018 Feb 14.
PubMed 29517971Sahu S, Lata I, Singh S, Kumar M. Revisiting hemophilia management in acute medicine. J Emerg Trauma Shock. 2011 Apr;4(2):292-8. doi: 10.4103/0974-2700.82225.
PubMed 21769217Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, Ludlam CA, Mahlangu JN, Mulder K, Poon MC, Street A; Treatment Guidelines Working Group on Behalf of The World Federation Of Hemophilia. Guidelines for the management of hemophilia. Haemophilia. 2013 Jan;19(1):e1-47. doi: 10.1111/j.1365-2516.2012.02909.x. Epub 2012 Jul 6.
PubMed 22776238Mehta P, Reddivari AKR. Hemophilia. 2023 Jun 5. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2026 Jan-. Available from http://www.ncbi.nlm.nih.gov/books/NBK551607/
PubMed 31869071Srivastava A, Santagostino E, Dougall A, Kitchen S, Sutherland M, Pipe SW, Carcao M, Mahlangu J, Ragni MV, Windyga J, Llinas A, Goddard NJ, Mohan R, Poonnoose PM, Feldman BM, Lewis SZ, van den Berg HM, Pierce GF; WFH Guidelines for the Management of Hemophilia panelists and co-authors. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020 Aug;26 Suppl 6:1-158. doi: 10.1111/hae.14046. Epub 2020 Aug 3. No abstract available.
PubMed 32744769
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Emicizumab Treatment on the Musculoskeletal System in Children With Hemophilia A: A Prospective Study
- ClinicalTrials.gov ID
- NCT07190118
- Acronym / sponsor ID
- 2025/096
- Keywords
- Hemophilia A, Emicizumab, Musculoskeletal system, Measurement
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Hasan Kalyoncu UniversityUniversity, hospital or other
- Study start
- 30 Sep 2025 (estimated)
- Primary completion
- 10 Nov 2025 (estimated)
- Study completion
- 10 Feb 2026 (estimated)
- First posted
- 24 Sep 2025 (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.
The aim of our study is to investigate the effects of emicizumab treatment on the musculoskeletal system in children with hemophilia A.
Detailed description
Hemophilia is a rare inherited coagulation disorder resulting from a deficiency of factor (F) VIII or IX. It is a chronic group of diseases that primarily manifests as intra-articular (hemarthrosis) and intramuscular (hematoma) bleeding and affects quality of life. Easy ecchymosis formation in early childhood, especially spontaneous intra-articular and intramuscular bleeding, and a history of bleeding lasting longer than expected after interventions and trauma should raise the suspicion of hemophilia. The severity of bleeding findings is directly related to the degree of FVIII or FIX deficiency. While the clinical picture varies depending on the duration and frequency of bleeding, the severity of the disease is classified as severe (<1% IU dL-1), moderate (1%-5% IU dL-1), and mild (5%-40% IU dL-1). Hemophilia A occurs in 85% of the population (1 in 5,000 male births), while this rate is 15% in Hemophilia B (1 in 30,000 male births). Treatment of hemophilia is primarily divided into treatment of acute bleeding and prophylaxis. The primary goal in the treatment of acute bleeding is to achieve hemostasis within the first two hours after the onset of symptoms. Patients often recognize a bleeding episode by a tingling sensation or an "aura" before the bleeding begins. In such cases, factor replacement therapy should be administered immediately. In a patient presenting with severe acute bleeding, after determining the location and severity of the bleeding, factor VIII or factor IX and high-dose clotting factor concentrate (CFC) are administered. Factor concentrate doses should be 50 IU/kg factor VIII or 100-120 IU/kg factor IX. Even if bleeding slows or stops, CFC should be administered to maintain healing. Frequent measurement of factor levels should be performed to ensure that desired levels are maintained. Acetylsalicylic acid (ASA) and nonsteroidal anti-inflammatory drugs (NSAIDs) should be avoided during pain management due to their effects on platelet function and increased risk of bleeding. Prophylaxis treatment includes treating acute bleeding as well as replacing the missing factor in cases where there is no bleeding. Prophylactic treatment has many advantages. Prophylaxis is generally initiated at an early age to prevent and reduce the risk of joint bleeding and the development of hemophilic arthropathy. Dosing is personalized and adjusted according to the severity of bleeding. It protects joint health by reducing hemarthrosis episodes and reduces the need for joint surgical interventions. Prophylactic factor replacement in hemophilia treatment is administered two or three times a week via intravenous infusion. Emicizumab, one of the alternative pharmacological treatments developed in recent years, is a subcutaneous treatment method that provides patients with access to the medication within clinical and financial healthcare services in Turkey as of May 2025. Emicizumab, Fitusiran, and Concizumab, both inhibit natural anticoagulant pathways. With these subcutaneous treatments, injection frequency can vary depending on the molecule, but can be daily, weekly, or monthly. One of the most important advantages of these treatments is their effectiveness in the presence of alloantibodies, which are a significant cause of morbidity and mortality in hemophilia. Treatment features such as reduced injection frequency of factor replacements administered subcutaneously, eliminating the need for intravenous infusions, and increasing physical activity opportunities are thought to produce significant benefits affecting quality of life, in addition to controlling and preventing bleeding. Therefore, new drugs that promise less bleeding with fewer and more comfortable injections are quite attractive. It is believed that reduced bleeding will equally reduce musculoskeletal damage, support improved joint health, and increase the individual's level of functionality, resulting in improved quality of life by ensuring independence in daily life. Improved musculoskeletal health and independence in daily life will increase children's exercise habits. The positive contributions to the musculoskeletal system, such as increased overall muscle mass and maintenance of joint health, will also increase the effectiveness of physiotherapy programs. There are no other studies examining the effects of this newly introduced drug on the musculoskeletal system in children in Turkey. Therefore, the aim of our study was to investigate the effects of emicizumab treatment on the musculoskeletal system in children with hemophilia A.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Target follow-up
- 6 Months
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A Group | – | –This will be the first assessment before the child begins treatment with emicizumab. During the first month of treatment, the child and parents will be asked to note any joint or muscle bleeding, including the bleeding site, duration of bleeding, and whether hospitalization is necessary. At the end of the first month, parents will be contacted by phone to assess bleeding episodes, and any bleeding that occurred during the first month will be recorded. The same assessments will then be repeated at the third and sixth months of treatment. The effectiveness of the treatment will be assessed after the first six months through an analysis of the assessments. |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Male patients under 18 years of age diagnosed with hemophilia A will constitute the study population.
Key inclusion criteria (6 listed)
- Children who are under 18 years of age,
- Have been diagnosed with Hemophilia A by a physician,
- Have a factor level of 1% (IU) or less,
- Have inhibitors positive or negative,
- Have no obstacles to starting subcutaneous (emicizumab) treatment,
Key exclusion criteria (1 listed)
- Children with a history of cerebrovascular bleeding who present any neurological findings that would prevent participation in the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
The pain
Time frame: through of the study, average 6 months
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.
Hemophilia Joint Health Score (HJHS)
Time frame: through of the study, average 6 months
Hemophilia Joint H is a scoring system that targets the evaluation of three major joints (elbow, knee, and ankle). Consists of a total of 9 questions: 8 questions assessing joints and 1 question providing a global gait score. The hemophilia joint health score is calculated by assessing joint swelling, the longevity of the swelling, the presence of atrophy in the surrounding soft tissue, crepitus during active movement, loss of flexion or extension, pain, muscle strength, and the total global gait score. Hemophilia Joint Health Score values range from 0 to 124, with higher scores indicating poorer joint health.
Lower extremity functionality and mobility- 5-Time Sit-to-Go Test
Time frame: through of the study, average 6 months
The 5-time sit-to-stand test is scored based on the time (in seconds to the nearest decimal place) in which a patient can move from a sitting position to a standing position and back to a sitting position five times. At the beginning of the test, the test participant is instructed to sit in a chair with their back straight. They are also instructed to fold their arms across their chest. The test participant should then be instructed to sit and stand five times as quickly as possible. The shorter the time taken to complete the test, the better the test result.
Upper extremity functionality
Time frame: through of the study, average 6 months
The Questionnaire for Arm, Shoulder, and Hand Disabilities (Q-DASH) will be used to assess individuals' upper extremity functions. This questionnaire is a self-administered measurement tool with validated validity and reliability in Turkish. The questionnaire, consisting of 30 questions, assesses the individual's ability to perform functional activities (21 items), pain (5 items), and psychosocial aspects of the disease (4 items). The total score ranges from 0 to 100, with a higher score indicating better outcomes.
Static balance
Time frame: through of the study, average 6 months
The One-Legged Standing Test will be administered to assess patients' static balance. In the one-leg standing test, the subject will stand with their arms hanging at their sides and lift each lower extremity one leg at a time, measuring the duration of their stance. The time will be stopped if they reposition their supporting leg, touch the ground with their raised foot, or seek support from an observer or any other support.
Lower extremity functionality and mobility- 30-second Sit-to-Go Test
Time frame: through of the study, average 6 months
The 30-Second Chair Test is administered using a folding chair without arms. The chair, with rubber feet, is placed against a wall to prevent movement. The participant sits in the center of the chair, back straight, feet approximately shoulder-width apart and slightly behind the knees, with one foot slightly in front of the other to assist with balance. Arms are crossed at the wrists and held toward the chest. The participant is encouraged to perform as many complete poses as possible in 30 seconds. The participant is asked to sit fully between each pose. The tester, monitoring the participant's performance to ensure proper form, silently counts the number of each pose. The score is the total number of poses performed in the 30 seconds (more than half the poses by the end of 30 seconds are considered complete poses).
Lower extremity functionality and mobility- Timed Up-and-Go Test
Time frame: through of the study, average 6 months
The Timed Up and Go Test (TUG) is a quick and easy-to-administer clinical test that assesses an individual's basic mobility skills. The test involves the patient rising from a standard chair, walking a distance of 3 meters (10 feet), turning around, returning to the chair, and sitting down again. The time taken to complete this task is recorded in seconds.
3 secondary outcomes
Fear of movement
Time frame: through of the study, average 6 months
Functional independence levels
Time frame: through of the study, average 6 months
Children's Quality of Life
Time frame: through of the study, average 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2025.
- Results publication: PubMed 16236109Poonnoose PM, Manigandan C, Thomas R, Shyamkumar NK, Kavitha ML, Bhattacharji S, Srivastava A. Functional Independence Score in Haemophilia: a new performance-based instrument to measure disability. Haemophilia. 2005 Nov;11(6):598-602. doi: 10.1111/j.1365-2516.2005.01142.x.
- Results publication: PubMed 23422454Kumban W, Amatachaya S, Emasithi A, Siritaratiwat W. Five-times-sit-to-stand test in children with cerebral palsy: reliability and concurrent validity. NeuroRehabilitation. 2013;32(1):9-15. doi: 10.3233/NRE-130818.
- Results publication: PubMed 30295404McLaughlin P, Morris R, Chowdary P. Investigating the relationship between the HJHS and HAL in routine clinical practice: A retrospective review. Haemophilia. 2018 Nov;24(6):988-994. doi: 10.1111/hae.13614. Epub 2018 Oct 8.
- Results publication: PubMed 29485331R CB, A TO, S PA, J CJ, F Q, J NM, Ja LP. Using the Hemophilia Joint Health Score for assessment of children: Reliability of the Spanish version. Physiother Theory Pract. 2019 Apr;35(4):341-347. doi: 10.1080/09593985.2018.1443356. Epub 2018 Feb 27.
- Results publication: PubMed 24330460Sun J, Hilliard PE, Feldman BM, Zourikian N, Chen L, Blanchette VS, Luke KH, Poon MC. Chinese Hemophilia Joint Health Score 2.1 reliability study. Haemophilia. 2014 May;20(3):435-40. doi: 10.1111/hae.12330. Epub 2013 Dec 16.
- Results publication: PubMed 16919083Hilliard P, Funk S, Zourikian N, Bergstrom BM, Bradley CS, McLimont M, Manco-Johnson M, Petrini P, van den Berg M, Feldman BM. Hemophilia joint health score reliability study. Haemophilia. 2006 Sep;12(5):518-25. doi: 10.1111/j.1365-2516.2006.01312.x.
- Results publication: PubMed 17106618Yakut Y, Yakut E, Bayar K, Uygur F. Reliability and validity of the Turkish version short-form McGill pain questionnaire in patients with rheumatoid arthritis. Clin Rheumatol. 2007 Jul;26(7):1083-7. doi: 10.1007/s10067-006-0452-6. Epub 2006 Nov 15.
- Results publication: PubMed 28851125Steen Carlsson K, Andersson E, Berntorp E. Preference-based valuation of treatment attributes in haemophilia A using web survey. Haemophilia. 2017 Nov;23(6):894-903. doi: 10.1111/hae.13322. Epub 2017 Aug 29.
- Results publication: PubMed 27913543Meeks SL, Batsuli G. Hemophilia and inhibitors: current treatment options and potential new therapeutic approaches. Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):657-662. doi: 10.1182/asheducation-2016.1.657.
- Results publication: PubMed 29517971Franchini M, Mannucci PM. Non-factor replacement therapy for haemophilia: a current update. Blood Transfus. 2018 Sep;16(5):457-461. doi: 10.2450/2018.0272-17. Epub 2018 Feb 14.
- Results publication: PubMed 21769217Sahu S, Lata I, Singh S, Kumar M. Revisiting hemophilia management in acute medicine. J Emerg Trauma Shock. 2011 Apr;4(2):292-8. doi: 10.4103/0974-2700.82225.
- Results publication: PubMed 22776238Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, Ludlam CA, Mahlangu JN, Mulder K, Poon MC, Street A; Treatment Guidelines Working Group on Behalf of The World Federation Of Hemophilia. Guidelines for the management of hemophilia. Haemophilia. 2013 Jan;19(1):e1-47. doi: 10.1111/j.1365-2516.2012.02909.x. Epub 2012 Jul 6.
- Individual participant dataSharing planned: No
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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.
Tuğba GÖNEN, Asisst. Prof. Dr. principal investigator
Hasan Kalyoncu University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A and Musculoskeletal Assessment, 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 1 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: opening soon, as of the sponsor's last update on 24 Sep 2025. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Tuğba GÖNEN, Asisst. Prof. Dr. (505 090 58 46, tugba.badat@hku.edu.tr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The pain (time frame: through of the study, average 6 months). The study lists 7 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 NCT07190118, which also lists 14 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 24 Sep 2025.