Early Check: Expanded Screening in Newborns

Study Identifier:
18-0009
ClinicalTrials.gov Identifier:
NCT03655223
EudraCT Identifier:
N/A
Sponsor:
RTI International
Collaborator:
University of North Carolina, Chapel Hill,The John Merck Fund,Duke University,Wake Forest University,North Carolina Department of Health and Human Services,National Center for Advancing Translational Sciences (NCATS),Cure SMA,The National Fragile X Foundation,Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD),Asuragen, Inc.,Sarepta Therapeutics, Inc.,Muscular Dystrophy Association,The Leona M. and Harry B. Helmsley Charitable Trust,Juvenile Diabetes Research Foundation,Janssen Pharmaceuticals,GeneDx,Illumina, Inc.
Study Contact Information:
N/A
Recruiting

Study Details

Medical Condition
  • Unmapped
  • Ignored
  • Hemophilia
  • Hemophilia A
  • Hemophilia B
  • Von Willebrand Disease
  • Factor VII Deficiency
Study Drug
    Date
    Oct 2018 - Nov 2025
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Female & Male
    Age: 1 Day - 31 Days years
    Requirements Information

    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
    RTI International
    Research Triangle Park, North Carolina, United States, 27709
    Status
    N/A