- 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 - 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.
Netherlands · Germany - 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
Belgium · Netherlands · Germany