Effects of Emicizumab Treatment in Patients With Hemophilia A

Launched by HASAN KALYONCU UNIVERSITY · Sep 23, 2025

Keywords

ClinConnect Summary

This is an observational study from Hasan Kalyoncu University in Gaziantep, Turkey, looking at how emicizumab—a medicine given under the skin to prevent bleeding—affects the muscles and joints of children with Hemophilia A. The study plans to enroll about 20 boys under 18 who have Hemophilia A (severe cases are more common) and who can start emicizumab. It will record their bleeding episodes and joint health before starting treatment and then again at 1, 3, and 6 months after beginning the medicine. The goal is to understand whether emicizumab helps improve pain, joint function, balance, and overall daily life.

Participants will undergo a series of simple tests and questionnaires to measure pain, how well joints and limbs work, balance and mobility, and quality of life. Specific measures include joint health scores, several performance tests (like sit-to-stand and gait tests), and surveys about fear of movement, independence in daily activities, and quality of life for both children and their parents. The study is not recruiting yet, with an estimated start date of September 30, 2025, and a projected completion around February 10, 2026. If you’re seeking more details or have questions, the study team can be reached at Hasan Kalyoncu University.

Gender

MALE

Eligibility criteria

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