Severe Congenital Hemostatic Defects, Cerebral MIcrobleeds and COGnition

Study Identifier:
2022_0601
ClinicalTrials.gov Identifier:
NCT06090201
EudraCT Identifier:
N/A
Will Be Recruiting

Study Details

Medical Condition
  • Hemophilia
Study Drug
    Date
    Nov 2023 - May 2026
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Female & Male
    Age: 18+ years years
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • Inclusion Criteria:
    • Male or female, older than 18 years old, no upper age limit
    • Adult patients with a severe congenital haemostatic defect
    • Severe or moderate congenital haemophilia A (or B) defined as \<5 IU/dL (\<5%) endogenous FVIII (FIX) activity at screening
    • Severe von Willebrand disease defined as VWF: Act ≤15IU/dL (\<15%) at screening
    • Ability of the participant to provide signed and dated informed consent
    • Exclusion Criteria:
    • Contraindication for brain MRI
    • HIV infection to avoid a bias towards severe multifactorial neurological complications
    • Other known coagulation disorder(s) in addition to haemophilia or von Willebrand disease
    • Lack of informed consent

    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.