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)

Study Identifier:
300130
ClinicalTrials.gov Identifier:
NCT07582276
EudraCT Identifier:
N/A
Will Be Recruiting

Study Details

Medical Condition
  • Hemophilia A
Study Drug
    Date
    Jun 2026 - Aug 2031
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Male
    Age: 30 - 50 Years years
    Requirements Information
    Inclusion and Exclusion Criteria
    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.
    • 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.
    • History of bone fractures or surgical repair within 8 weeks prior to enrollment.
    • Participants with weight \>300 pounds, due to limitations of DEXA scanner

    Protocol Summary

    This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

    Trial Locations

    No locations found.