Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A

Study Identifier:
SPK-8011/8016-LTFU
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
Sponsor:
Spark Therapeutics, Inc.
Collaborator:
N/A
Study Contact Information:
N/A
Unmapped

Study Details

Medical Condition
  • Hemophilia A
Study Drug
    Date
    Aug 2018 - Dec 2032
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Male
    Age: 18+ years 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
    Boston Children's Hospital
    Boston, Massachusetts, United States, 02115
    Status
    N/A
    Location
    Mississippi Center for Advanced Medicine
    Madison, Mississippi, United States, 39110
    Status
    N/A
    Location
    Truman Medical Centers
    Kansas City, Missouri, United States, 64108
    Status
    N/A
    Location
    Oregon Health & Science University
    Portland, Oregon, United States, 97239
    Status
    N/A
    Location
    Pennsylvania State University Milton S. Hershey Medical Center
    Hershey, Pennsylvania, United States, 10733
    Status
    N/A
    Location
    The Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania, United States, 19104
    Status
    N/A