Bone health in severe hemophilia A: emicizumab vs efanesoctocog alfa

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This prospective observational study follows adults with severe hemophilia A on two different prophylaxis regimens to see how bone health changes over five years. Participants will have serial DXA scans, blood tests for bone remodeling and coagulation-related markers, and evaluations of joint health. The goal is to see if the choice of prophylaxis is associated with differences in bone mineral density and related factors.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. The participant or legally authorized representative is willing and able to provide written informed consent.
2. Diagnosis of severe hemophilia A (factor VIII activity \< 1%).
3. Male sex.
4. Age between 30 and 50 years (inclusive).
5. BMI between 18.5 and 40 kg/m2
6. The participant must have been on prophylaxis with Efanesoctocog alfa or Emicizumab for at least 3 months prior to enrollment and intend to remain on the current regimen for the next 5 years.
7. Willingness to undergo all research procedures, including DEXA scans and the collection of blood samples.
8. Willingness to complete all standard-of-care bleeding and treatment logs.

Exclusion Criteria:

1. Unwillingness of the participant, parent, or legally authorized representative to provide informed consent.
2. Diagnosis of a bleeding disorder other than or in addition to severe hemophilia A.
3. Active Factor VIII inhibitors at the time of enrollment
4. History of a disease known to influence bone metabolism unrelated to a bleeding disorder. (Examples: Paget's disease, osteogenesis imperfecta, Ehlers Danlos syndrome, Hyperparathyroidism)
5. Past or present treatment with any anti-osteoporotic medication, excluding oral vitamin D or oral calcium supplements.
6. Documented HIV infection or HCV infection (whether in progress or cured) at the cirrhotic stage.
7. Presence of a non-removable metal device that would interfere with research procedures.
8. Inability to tolerate a DEXA scan due to limited range of motion or body habitus.
9. History of bone fractures or surgical repair within 8 weeks prior to enrollment.
10. Participants with weight \>300 pounds, due to limitations of DEXA scanner

Where it's running

• Arkansas Children's
  Little Rock, Arkansas, United States Recruiting

Study contacts

• Principal investigator: Divyaswathi Citla Sridhar, MD — Arkansas Children's Reserach Institute
• Study coordinator: Carol D Pierce, RN
• Email: piercecarold@uams.edu
• Phone: 501-364-4440

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.