A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

Study Identifier:
R131L1265-HEMB-2318
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
Sponsor:
Regeneron Pharmaceuticals
Collaborator:
Intellia Therapeutics
Study Contact Information:
Recruiting

Study Details

Medical Condition
  • Hemophilia B
Study Drug
  • Drug: REGV131
  • Drug: LNP1265
Date
Sep 2024 - 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
Orthopaedic Hemophilia Treatment Center
Los Angeles, California, United States, 90007
Status
Recruiting
Location
Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting Disorders
New Orleans, Louisiana, United States, 70112
Status
Recruiting
Location
University of Michigan
Ann Arbor, Michigan, United States, 48109
Status
Recruiting
Location
McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario, Canada, L8N 3Z5
Status
Recruiting