A Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)
Starting soon
Conditions studied: Hemophilia a
In brief
This study will compare bone mineral density in patients with severe hemophilia A receiving prophylaxis with emicizumab or efanesoctocog alfa. Participants will undergo assessments of bone mineral density, bone remodeling biomarkers, thrombin generation, plasmin generation, and joint health over a five-year period. The study aims to evaluate whether differences in prophylactic therapy are associated with differences in bone health outcomes.
Key facts
- Study ID
- NCT07582276
- Run by
- Arkansas Children's Hospital Research Institute
- People needed
- 50
- Starts
- 2026-07-01
- Expected to finish
- 2032-08-01
- Last updated by the study team
- 2026-06-04
Who can join
Age: 30 and older, up to 50. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- The participant or legally authorized representative is willing and able to provide written informed consent.
- Diagnosis of severe hemophilia A (factor VIII activity < 1%).
- Male sex.
- Age between 30 and 50 years (inclusive).
- BMI between 18.5 and 40 kg/m2
- 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.
- Willingness to undergo all research procedures, including DEXA scans and the collection of blood samples.
- Willingness to complete all standard-of-care bleeding and treatment logs.
You may not qualify if…
- Unwillingness of the participant, parent, or legally authorized representative to provide informed consent.
- Diagnosis of a bleeding disorder other than or in addition to severe hemophilia A.
- Active Factor VIII inhibitors at the time of enrollment
- History of a disease known to influence bone metabolism unrelated to a bleeding disorder. (Examples: Paget's disease, osteogenesis imperfecta, Ehlers Danlos syndrome, Hyperparathyroidism)
- Past or present treatment with any anti-osteoporotic medication, excluding oral vitamin D or oral calcium supplements.
- Documented HIV infection or HCV infection (whether in progress or cured) at the cirrhotic stage.
- Presence of a non-removable metal device that would interfere with research procedures.
- Inability to tolerate a DEXA scan due to limited range of motion or body habitus.
- History of bone fractures or surgical repair within 8 weeks prior to enrollment.
- Participants with weight >300 pounds, due to limitations of DEXA scanner
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.