A Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Fa… (NCT07582276) | Clinical Trial Compass
Not Yet RecruitingNot Applicable
A Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)
50 participantsStarted 2026-07-01
Plain-language summary
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.
Who can participate
Age range
30 Years – 50 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Longitudinal change in femoral neck bone mineral density (g/cm²)
Timeframe: Baseline and annually through 5 years.
Trial details
NCT IDNCT07582276
SponsorArkansas Children's Hospital Research Institute
. Willingness to complete all standard-of-care bleeding and treatment logs.
Exclusion criteria
. 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.