Von Willebrand Disease

Researchers are conducting the EMPOWER trial, a pilot multi-center, placebo-controlled, double-blind, crossover randomized trial lasting two years. It focuses on female outpatients with von Willebrand disease VWD who experience heavy menstrual bleeding HMB. The study aims to assess whether this trial design is feasible and viable, and to explore the sensitivity of clinical outcomes to guide a future definitive trial. Participants will be randomly assigned to receive either a plasma-derived von Willebrand factorFactor VIII concentrate called Wilate4 or a placebo normal saline, both given with standard care. Treatment is provided over four menstrual cycles during the first period, followed by a one-cycle washout without study treatment. Then, participants switch to the other treatment in the second period. Wilate4 is administered intravenously at doses of 30-60 IU VWFRCokg on the two heaviest bleeding days within the first four days of menstruation, with optional additional doses. During the study, participants receive infusions by a nurse and use specific feminine hygiene products supplied by the sponsor. Researchers will evaluate trial feasibility by measuring participant retention, blinding success, and data completion. They will also assess menstrual bleeding severity using a modified pictorial blood assessment chart mPBAC and monitor clinical outcomes like bleeding events, hemoglobin and ferritin levels, fatigue, and adverse reactions. The total study participation is two years, including both treatment periods and washout.

Researchers are studying HMB-002 in people with Von Willebrand Disease VWD to evaluate its safety, tolerability, how the body processes the drug, its effects, and preliminary efficacy. This first-in-human Phase 12 trial includes three parts to explore different dosing schedules and combinations with standard factor concentrates for various VWD types. The study aims to understand how HMB-002 works alone and alongside standard treatments in participants aged 16 to under 70 years. The study is divided into three parts Part A tests single ascending doses of HMB-002 administered under close monitoring to assess safety and drug behavior over about 12 weeks. Part B evaluates repeated doses over approximately 21 weeks to assess safety and initial effects on bleeding events. Part C studies a single dose of HMB-002 given with a regular factor concentrate dose in participants who already receive this treatment, lasting about 17 weeks. Dosing schedules in Part B depend on Part A results. Participants will be monitored closely throughout the study with safety assessments, blood tests, and evaluations of bleeding rates and factor activity from Day 1 up to Day 113. Researchers will track any treatment-emergent adverse events and measure pharmacokinetic parameters like maximum drug concentration and elimination half-life. They will also assess pharmacodynamic markers such as von Willebrand factor antigen and activity, and factor VIII activity. Overall participation lasts up to about 17 to 21 weeks depending on the study part.

Researchers are studying people with Von Willebrand Disease VWD, focusing on how often bleeding events happen, their impact on quality of life, and the social and clinical effects of bleeds. This screening study gathers baseline data on bleeding and treatment rates in participants with VWD, mainly Type 1, but may include Types 2 and 3 with sponsor approval. The findings will help compare outcomes in future clinical studies like Velora Pioneer. The study involves screening and a baseline check, followed by an observation period of about 4 months during which participants will have telemedicine check-ins every two weeks. These check-ins monitor bleeding events and treatment use through a bleed diary. Participants can opt to extend this observation period up to 12 months if they wish. Participants will provide information about their bleeding events and treatments during the study. Researchers will assess bleeding rates, hospital stays, and quality of life using questionnaires and clinical evaluations. Outcomes measured include annual bleeding rates, treatment use, iron status, and bleeding severity scores. The study concludes with data collection over the observation period, helping to understand the experience of people with VWD over time.

This research aims to evaluate the safety and effectiveness of a medical device called ETHIZIA compared to SURGICEL Original for controlling minimal, mild, or moderate soft tissue bleeding during open surgery. The study focuses on bleeding that occurs in areas such as the abdomen, pelvis, thorax excluding the heart, and extremities, where standard methods to stop bleeding are ineffective or impractical. Participants will be randomly assigned to receive either the ETHIZIA patch or SURGICEL Original applied directly to the bleeding site during surgery. Both devices are intended to stop bleeding by achieving hemostasis within 3 minutes and without rebleeding up to 10 minutes after application. After the surgery, participants will be monitored and followed up for 28 days. During the study, participants will undergo assessments to measure the percentage of cases achieving hemostasis at the target bleeding site and the time taken to stop bleeding. Additional evaluations include monitoring for rebleeding, treatment failures, and the need for additional applications or surgical maneuvers. Safety and efficacy data will be collected up to 10 minutes intraoperatively and through follow-up visits within 28 days after surgery.

Researchers are conducting a Phase III, multicenter, open-label clinical study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and older diagnosed with Type 3 von Willebrand disease VWD. The study compares emicizumab prophylaxis with standard of care SOC on-demand therapy in participants previously on on-demand treatment, and assesses emicizumab prophylaxis in those already on SOC prophylactic therapy by comparing with prior data from a non-interventional study. Participants are divided into three groups Arm A receives emicizumab subcutaneous injections weekly for 4 weeks followed by doses every 2 weeks as prophylaxis Arm B continues their current SOC on-demand therapy for 24 weeks and Arm C, previously on SOC prophylaxis, receives emicizumab prophylaxis. After completing 24 weeks of treatment, participants benefiting from emicizumab in Arms A and C may continue the drug in an extension phase. Participants in Arm B may switch to emicizumab prophylaxis after 24 weeks. During the study, participants will have their bleeding rates measured and compared between treatments, along with monitoring for adverse events, thromboembolic events, injection-site reactions, and laboratory abnormalities. Various health parameters including vital signs, electrocardiograms, and patient-reported outcomes like pain and fatigue will be assessed regularly. The study duration may extend up to nearly four years from first dose through follow-up and extension periods.

Researchers are evaluating the safety, tolerability, and best dose for Phase 2 of KLN-1010, a new gene therapy, in patients with relapsed or refractory multiple myeloma. This treatment aims to generate anti-B cell maturation antigen Anti-BCMA CAR-T cells directly in the body to target cancer cells. The trial is a Phase 1 study sponsored by Kelonia Therapeutics, Inc. and focuses on patients who have received multiple prior therapies. Participants receive a single specified dose of KLN-1010 during the treatment phase. The study follows them for up to 15 years after dosing to monitor safety and establish the recommended dose for future testing. Pharmacokinetics, including how the drug and CAR-T cells behave in the body, are assessed for up to two years. Disease status is also tracked from dosing until progression or for up to 15 years. Throughout the study, participants undergo regular evaluations including safety assessments for adverse events and dose-limiting toxicities. Laboratory tests and clinical assessments measure how the therapy affects the disease and the participants health. Long-term monitoring ensures ongoing safety and effectiveness measurements, with the entire participation lasting up to 15 years after treatment.

Researchers are evaluating the safety, pharmacokinetics, pharmacodynamics, and preliminary effectiveness of an anti-GPRC5D CAR-T cell product called OriCAR-017 in adults with relapsed or refractory multiple myeloma. This Phase III open-label study is the first clinical trial of OriCAR-017 in the United States by OriCell Therapeutics Co., Ltd., aiming to find suitable dosing and assess early treatment results in this patient group. The study includes a Phase I dose escalation stage with three different doses given as a single intravenous infusion to up to 18 participants. This is followed by a dose expansion stage with 10-15 participants and then a Phase II stage that may include up to 48 participants. Each participant receives one infusion of OriCAR-017 to evaluate its effects and safety. Participants will be closely monitored for up to two years after treatment. Researchers will assess the maximum tolerated dose and dose-limiting toxicities within 28 days after infusion. They will also study how the drug moves through and affects the body, measure response duration, progression-free survival, overall survival, and other response rates. Regular evaluations include laboratory tests, clinical assessments, and safety monitoring throughout the study period.

Researchers are investigating a new drug called BAY 3389934 to find a better way to treat people with sepsis-induced coagulopathy, a serious condition where an active immune response causes uncontrolled blood clotting, damaging blood vessels and organs. This early-phase study aims to learn about the safety, appropriate dose, and effects of BAY 3389934 in patients receiving treatment for this condition in intensive care units. The research is focused on monitoring medical problems, called adverse events, that occur during and after treatment. Participants will receive BAY 3389934 as a continuous intravenous infusion for up to 96 hours. They will be divided into two groups the first group will receive a low starting dose, and their response will be closely monitored to adjust the dose if needed. If there are no serious side effects, the second group will receive a higher dose. This dose escalation approach helps determine the best dose for future studies. During the approximately 28-day study, participants will have blood and urine samples taken, physical exams, vital signs checked, and heart health monitored with electrocardiograms. Researchers will track the number and severity of any treatment-emergent adverse events within about four days after starting the infusion. They will also measure blood clotting times over the first six days to assess effects on coagulation. The study is designed to carefully observe safety and drug effects while patients receive intensive care.

This research aims to assess the effectiveness and safety of adding oral anticoagulation OAC to background antiplatelet therapy in patients who develop new-onset post-operative atrial fibrillation POAF after isolated coronary artery bypass graft CABG surgery. The study is a prospective, multicenter, open-label, randomized trial comparing OAC with no OAC to evaluate the prevention of thromboembolic events and the risk of major bleeding. Participants are randomly assigned to either an OAC-based strategy using vitamin K antagonists or approved direct oral anticoagulants alongside antiplatelet therapy, or to an antiplatelet-only strategy. The anticoagulation treatment lasts for 90 days, with option for crossover to OAC if recurrent atrial fibrillation occurs after 30 days in the control group. Up to 500 patients may also participate in a digital health substudy using a wearable heart rhythm monitor for 30 days post-discharge. During the study, participants have follow-up visits at 30, 60, 90, and 180 days after randomization, including phone contacts and clinical assessments. Researchers will monitor outcomes such as death, stroke, transient ischemic attacks, myocardial infarction, thromboembolism, and bleeding events up to 180 days. Data from patients who decline randomization are collected in a parallel registry to compare baseline risk and treatment strategies.

Researchers are studying how cardiovascular surgeons use hemostatic agents during heart and blood vessel surgeries. These agents help control bleeding and affect outcomes like blood loss, transfusions, surgery time, and complications. The study aims to understand real-world use, how well practice matches guidelines, and differences between what surgeons think they know and how they actually decide on treatments. It is an observational study conducted at a single hospital in Milan, Italy. The study involves an anonymous survey completed by cardiac and vascular surgeons and a review of hospital records on the use of hemostatic agents. Surgeons answer clinical vignettes to assess their decision-making compared to current guideline recommendations. The study measures the appropriateness of agent selection using a Knowledge-Practice Alignment Score KPA-Score. It also looks at usage patterns, barriers to proper use, and alignment between reported practices and actual consumption. Participants are surgeons involved in cardiovascular operations who complete the survey anonymously. Hospital data on hemostatic agent use is analyzed at the unit level without patient identifiers. Researchers collect information through questionnaires, clinical cases, and pharmacy records. The main outcome is the proportion of surgeons with a KPA-Score of 60% or higher, indicating appropriate selection. Secondary outcomes include knowledge perception, practice patterns, and barriers. The study runs from July 2026 to August 2026.