- ongoing, recruitingTherapeutic confirmatory (Phase III)
Phase III, open-label, single-dose, multi-center multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene (AAV5-hFIXco-Padua, AMT-061) administered to adult subjects with severe or moderately severe hemophilia B
This study is testing a new gene therapy called Hemgenix for men with severe or moderately severe haemophilia B. It compares the number of bleeding episodes in patients receiving Hemgenix to those on standard treatment, over roughly a year and a half.
Belgium · Netherlands · Germany - ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humans
A Phase I/IIb extension study assessing the long-term safety and efficacy of an adeno-associated viral vector containing a codon-optimized human factor IX gene (AAV5-hFIX) previously administered to adult patients with severe or moderately severe haemophilia B during the CT-AMT-060-01 Phase I/II study.
This study is looking at the long-term safety and how well a gene therapy called Hemgenix works for men with severe or moderately severe haemophilia B. It follows up on past participants to see the effects over 6 to 10 years.
Netherlands · Germany - enrolling by invitation
A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN
This study looks at how well Refixia/REBINYN works and if it has any side effects for males with Haemophilia B over a long time. Participants will receive their usual treatment and share information about their health, bleeds, and quality of life.
Austria · Belgium · Canada - enrolling by invitation
Adverse Event Data Collection From External Registries on Nonacog Beta Pegol
This study looks at safety information from existing patient records for people with haemophilia B who have been treated with a medicine called nonacog beta pegol. Researchers are gathering information from large databases like PedNet and EUHASS to better understand any side effects from this treatment.
Netherlands · United Kingdom - active not recruiting
Observatory of Patients With Haemophilia B Treated by IdElvion®
This study looks at how a medicine called Idelvion® works for people in France with haemophilia B in their everyday lives. It aims to confirm it's effective and safe, building on earlier research.
France