Treatment

Hemgenix 1 x 10^13 genome copies/mL concentrate for solution for infusion clinical trials

2 indexed studies · 0 currently recruiting

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.
For: Haemophilia B
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
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.
For: Haemophilia B
Belgium · Netherlands · Germany