Gene Therapy Study in Severe Haemophilia A Patients (270-201)

Study Identifier:
BMN 270-201
ClinicalTrials.gov Identifier:
EudraCT Identifier:
Sponsor:
BioMarin Pharmaceutical
Collaborator:
N/A
Study Contact Information:
N/A
Recruitment Complete

Study Details

Medical Condition
  • Hemophilia A, Severe
Study Drug
  • Biological: valoctocogene roxaparvovec
Date
Aug 2015 - Mar 2024
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
Queen Elizabeth Hospital Birmingham
Birmingham, United Kingdom
Status
N/A
Location
Addenbrooke's Hospital
Cambridge, United Kingdom
Status
N/A
Location
St. Thomas' Hospital
London, United Kingdom
Status
N/A
Location
The Royal London Hospital
London, United Kingdom
Status
N/A
Location
University Hospital Southampton NHS Foundation Trust
Southampton, United Kingdom
Status
N/A