Palliative Care Needs of Children With Rare Diseases and Their Families

Study Identifier:
R01HD117137-01
ClinicalTrials.gov Identifier:
NCT06938542
EudraCT Identifier:
N/A
Will Be Recruiting

Study Details

Medical Condition
  • Unmapped
  • Hemophilia A, Severe
Study Drug
    Date
    Feb 2026 - Feb 2029
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Female & Male
    Age: 12 Months - 99 Years years
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • Child inclusion criteria:
    • ≥1.0 years and \<18.0 years at enrollment.
    • Unable to participate in end-of-life care decision-making.
    • Has a rare disease as operationally defined by NIH's Genetic and Rare Diseases Information Center (GARD).
    • Not under a Do Not Resuscitate Order or Allow a Natural Death Order.
    • Not in the Intensive Care Unit.
    • Family caregiver inclusion criteria:
    • \> 18.0 years at enrollment.
    • Child's family caregiver/legal guardian.
    • Not known to be developmentally delayed.
    • Support person inclusion criteria:
    • \> 18.0 years at enrollment.
    • Chosen by family caregiver.
    • Not known to be developmentally delayed.
    • Exclusion Criteria:
    • Family caregiver or support person is actively homicidal, suicidal, or psychotic at the time of enrollment.

    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

    Location
    Status
    Location
    Children's National Hospital
    Washington D.C., District of Columbia, United States, 20010
    Status
    N/A