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The purpose of this study is to test the ability of an experimental drug (study drug) named VMX-C001 to bypass the effect of a medication that you are currently taking known as a FXa direct oral anticoagulant (FXa DOAC). FXa DOACs are anticoagulant (blood-thinning) drugs that reduce the risk of blood clots but increase the risk of bleeding. Therefore, in certain situations, patients taking FXa DOACs need to receive other drugs that can restore the ability of their blood to clot (coagulation), as in the cases of serious bleeding or when requiring urgent surgery or another procedure that is associated with a high risk of bleeding. Because you may have taken an FXa DOAC within the last 15 hours, it is usual for your medical team to want to restore coagulation during the surgery to prevent excessive bleeding. The main purpose of this research study is to: Evaluate the safety of the study drug, VMX-C001; Determine the efficacy of VMX-C001 (how well the drug works); Assess the pharmacokinetics (PK) of VMX-C001 (PK; how the drug is processed through your body); Assess the immunogenicity of VMX-C001 (how the drug works on your immune system).

This trial aims to investigate the use of Revumenib, a new drug being investigated for the treatment of specific types of Acute Myeloid Leukaemia (AML) in patients who are ineligible for intensive chemotherapy. It will look at whether the addition of Revumenib to the normal treatment for AML can improve the results of treatment. All participants on this trial will be treated with Azacitidine + Venetoclax which is the standard therapy for your specific type of AML. However, half of patients will randomly be assigned to receive an additional drug, Revumenib while the other half will only receive the standard treatment.

Study BRT-DA01-301 is a Phase 3, multicenter, randomized, sham surgery-controlled, double-blind study in approximately 102 participants with PD, of whom at least 30 are ?60 years of age at informed consent. Participants, care partners, investigators at the clinical sites (with the exception of pre-identified unblinded clinical site personnel), outcome assessors, care providers outside of the surgical team, and the sponsor (with the exception of pre-identified unblinded sponsor personnel) will be blinded to the study intervention.

Active, not recruiting This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B with inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups. In one group, participants will get study medicine from the start of the study. In the other group, participants will continue with their normal medicine and get study medicine after 6 months. Which treatment the participant gets is decided by chance. Participants who usually take medicine to prevent bleeds (prophylaxis treatment) or who are already being treated with concizumab (study medicine) will receive the study medicine from the start of the study. Participants will get 1 injection with the study medicine every day under the skin. These participants will have to do themselves and can be done at home. The study doctor will hand out the medicine in the form of a pen-injector. The pen-injector will contain the study medicine. The study will last for about seven years. The length of time the participants will be in the study depends on when they agreed to take part or when the medicine is available for purchase in their country (31 December 2026 at the latest). The time between visits will be approximately 4 weeks for the first 6 to 12 months, depending on the group participants are in and approximately 8 weeks for the rest of the study. Participants will be asked to record information into an electronic diary during the study and may also be asked to wear an activity tracker.