Bone Health in Severe Hemophilia A: Emicizumab Compared With Efanesoctocog Alfa
Compares bone density in adults with severe hemophilia A using emicizumab or efanesoctocog alfa.
Plain-language title generated from the registry record
Registry title: A Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)
Key facts
- Where
- Little Rock, United StatesSee the site
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 30 and 50 years old?
Source: NCT07582276, who can take part
Where in United States
- Arkansas Children'sLittle Rock, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Arkansas Children's Hospital Research Institute
- Trial code
- NCT07582276
- Record updated
- 5 October 2026
- Original description
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.Read full original descriptionShow less
A Multi-institution Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)
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.
Inclusion: 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.
Exclusion: Active Factor VIII inhibitors at the time of enrollment; History of bone fractures or surgical repair within 8 weeks prior to enrollment; Diagnosis of a bleeding disorder other than or in addition to severe hemophilia A; History of a disease known to influence bone metabolism unrelated to a bleeding disorder. (Examples: Paget's disease, osteogenesis imperfecta, Ehlers Danlos syndrome, Hyperparathyroidism); Documented HIV infection or HCV infection (whether in progress or cured) at the cirrhotic stage; Past or present treatment with any anti-osteoporotic medication, excluding oral vitamin D or oral calcium supplements; Unwillingness of the participant, parent, or legally authorized representative to provide informed consent; 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; Participants with weight >300 pounds, due to limitations of DEXA scanner.