Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

Study Identifier:
270-301
ClinicalTrials.gov Identifier:
EudraCT Identifier:
Sponsor:
BioMarin Pharmaceutical
Collaborator:
N/A
Study Contact Information:
N/A
Unmapped

Study Details

Medical Condition
  • Hemophilia A
Study Drug
  • Biological: valoctocogene roxaparvovec
Date
Dec 2017 - Nov 2020
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
Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Center
Los Angeles, California, United States, 90007-2664
Status
N/A
Location
UC Davis Hemophilia Treatment Center
Sacramento, California, United States, 95817
Status
N/A
Location
University of California San Diego, Hematology and Oncology, Hemophilia &Thrombosis Treatment Center
San Diego, California, United States, 92122
Status
N/A
Location
UCSF Medical Center
San Francisco, California, United States, 94143-0106
Status
N/A
Location
University of Colorado
Aurora, Colorado, United States, 80045
Status
N/A
Location
St. Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders
Tampa, Florida, United States, 33607
Status
N/A