Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

Study Identifier:
270-203
ClinicalTrials.gov Identifier:
EudraCT Identifier:
Sponsor:
BioMarin Pharmaceutical
Collaborator:
N/A
Study Contact Information:
N/A
Recruitment Complete

Study Details

Medical Condition
  • Hemophilia A
  • Ignored
Study Drug
  • Biological: Valoctocogene Roxaparvovec
Date
Apr 2018 - Nov 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
Kyung Hee University Hospital at Gangdong
Seoul, Republic of Korea
Status
N/A
Location
Severance Hospital, Yonsei University Health System
Seoul, Republic of Korea
Status
N/A
Location
Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center
Johannesburg, South Africa
Status
N/A
Location
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung, Taiwan, Province of China
Status
N/A
Location
Taichung Veterans General Hospital
Taichung, Taiwan, Province of China
Status
N/A
Location
National Taiwan University Hospital
Taipei, Taiwan, Province of China
Status
N/A