Effects of Emicizumab Treatment in Patients With Hemophilia A

NCT07190118 | Not Yet Recruiting DMC

• 1 other identifier: 2025/096
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of our study is to investigate the effects of emicizumab treatment on the musculoskeletal system in children with hemophilia A.

Conditions

Hemophilia A; Musculoskeletal Assessment; Musculoskeletal Abnormalities

Keywords

Hemophilia A; Emicizumab; Musculoskeletal system; Measurement

Outcome Measures

Primary Outcomes (7)

• 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.

  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.

  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.

  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.

  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.

  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).

  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.

  through of the study, average 6 months

Secondary Outcomes (3)

• Fear of movement

  through of the study, average 6 months

• Functional independence levels

  through of the study, average 6 months

• Children's Quality of Life

  through of the study, average 6 months

Study Arms (1)

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.

Eligibility Criteria

• Age: 1 Year - 18 Years
• Gender Eligibility Details: Because hemophilia A is an X-linked recessive disease, girls are carriers and boys are affected by the disease. Therefore, the study population consists of boys.
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Male patients under 18 years of age diagnosed with hemophilia A will constitute the study population.

You may qualify if:

• 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

You may not qualify if:

• \- Children with a history of cerebrovascular bleeding who present any neurological findings that would prevent participation in the study

Sponsors & Collaborators

• Hasan Kalyoncu University: lead

Study Sites (1)

Hasan Kalyoncu University

Gaziantep, Gaziantep, (505) 090-5846, Turkey (Türkiye)

Location

Related Publications (14)

• 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.

  PMID: 16236109 BACKGROUND

• Kumban 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.

  PMID: 23422454 BACKGROUND

• McLaughlin 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.

  PMID: 30295404 BACKGROUND

• R 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.

  PMID: 29485331 BACKGROUND

• Sun 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.

  PMID: 24330460 BACKGROUND

• Hilliard 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.

  PMID: 16919083 BACKGROUND

• Yakut 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.

  PMID: 17106618 BACKGROUND

• Steen 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.

  PMID: 28851125 BACKGROUND

• Meeks 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.

  PMID: 27913543 BACKGROUND

• Franchini 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.

  PMID: 29517971 BACKGROUND

• Sahu 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.

  PMID: 21769217 BACKGROUND

• Srivastava 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.

  PMID: 22776238 BACKGROUND

• Mehta P, Reddivari AKR. Hemophilia. 2023 Jun 5. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan-. Available from http://www.ncbi.nlm.nih.gov/books/NBK551607/

  PMID: 31869071 BACKGROUND

• Srivastava 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.

  PMID: 32744769 BACKGROUND

MeSH Terms

Conditions

Hemophilia A; Musculoskeletal Abnormalities

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Musculoskeletal Diseases; Congenital Abnormalities

Study Officials

• Tuğba GÖNEN, Asisst. Prof. Dr.

  Hasan Kalyoncu University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Tuğba GÖNEN, Asisst. Prof. Dr.

CONTACT

505 090 58 46; tugba.badat@hku.edu.tr

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Target Duration: 6 Months
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Asisst. Prof. Dr.

Study Record Dates

• First Submitted: September 1, 2025
• First Posted: September 24, 2025
• Study Start: September 30, 2025
• Primary Completion: November 10, 2025
• Study Completion: February 10, 2026
• Last Updated: September 24, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

TU (1)