Clinical trials

This is a randomised, actively controlled Phase IIb trial to evaluate the safety and pharmacodynamics (PD) of two blinded doses of subcutaneously administered vortosiran and open-label apixaban in patients with AF. The trial consists of three groups including 50 participants each. The participants will be randomized to either the low dose group or the high dose group receiving vortosiran or to the group receiving apixaban. The treatment will be open-label for vortosiran vs apixaban. The vortosiran dose will be blinded to patients and investigators. Both the low- and the high-dose group will receive two doses of vortosiran, the first dose at Day X and the second at Day Y. After 24 weeks, at the end of the vortosiran treatment period, the participants will start an open-label part of the trial and will be switched to standard-of-care apixaban treatment until Week 64 (End of Trial; EOT). Participants in the open-label apixaban arm will receive apixaban as standard of care during the entire trial.

Blood clots remain a serious cause of illness and death. Long-term treatment and prevention of these clots with blood thinners is often limited by bleeding risk, drug interactions, and daily dosing, leading many patients to stop treatment early. Both cancer and chemotherapy further increase the risk of clot formation. Vortosiran is an injectable therapy that lowers Factor XI (one of the body's natural clotting helpers) production in the liver, leading to a strong and long-lasting reduction in blood clotting activity with less risk of bleeding compared to current available treatments. Studies conducted so far have shown that vortosiran is safe and shows a dose-dependent Factor XI suppression (that is, a higher dose results in a higher level of suppression) that lasts for several months, supporting testing using a limited number of injections in patients who are at higher risk of blod clot formation. This study aims to study how safe and effective vortosiran is in patients who have had blood clots in the past and in cancer patients who are at high risk of developing blood clots.

The goal of this clinical trial is to learn if drug RBD5044 works to treat mixed dyslipidemia in adults. It will also learn about the safety of drug RBD5044. The main questions it aims to answer are: Does drug RBD5044 reduce the triglyceride levels? What medical problems may participants experience when taking drug RBD5044? Researchers will compare drug RBD5044 to a placebo to see if drug RBD5044 works to treat mixed dyslipidemia. Participants will: Receive RBD5044 or placebo twice during the trial (Day 1 and Day 84). Visit the clinic 11 times during 12 months (visit every 4-8 weeks) for checkups and tests.