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 factor:Factor 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 VWF:RCo/kg 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 evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of HMB-002 in adults aged 18 to 64 with Von Willebrand Disease (VWD), including Type 1, Type 1C, and Type 2A. This first-in-human, open-label Phase 1/2 study aims to understand how this investigational drug behaves in the body and its preliminary effects on bleeding episodes in affected participants. The study has two parts: Part A involves a single ascending dose of HMB-002 given subcutaneously to assess initial safety, tolerability, and drug behavior in the body over about 12 weeks. Part B involves multiple repeat doses, with dosing intervals determined after Part A results, lasting approximately 21 weeks. This phase evaluates safety and tolerability with repeated dosing and explores the drug's potential to reduce bleeding events. Participants will undergo regular monitoring including vital signs and laboratory tests to assess blood factors related to VWD and overall health. The study will track adverse events up to Day 113, measure drug levels and activity, and record bleeding rates. Participants must meet specific health criteria and agree to contraceptive measures if applicable. The total participation time varies by study part, with follow-ups and safety checks throughout the study period.

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.

Researchers are evaluating the safety and effectiveness of the ETHIZIA patch compared to SURGICEL Original for controlling minimal, mild, or moderate soft tissue bleeding during open surgeries. The study focuses on surgeries involving the abdomen, retroperitoneal area, pelvis, thoracic region (excluding heart surgery), and extremities. The goal is to see which device better achieves bleeding control within 3 minutes after application and prevents re-bleeding up to 10 minutes later. Participants will be randomly assigned to receive either the ETHIZIA patch or SURGICEL Original applied directly to bleeding soft tissue sites during surgery. Both treatments are applied intraoperatively at bleeding sites where conventional methods like sutures or cautery are not effective or practical. After surgery, participants will be followed for 28 days to monitor outcomes and any potential complications. During the study, researchers will closely monitor bleeding control at the target site, measuring the percentage of cases achieving hemostasis at 3 minutes and the absence of re-bleeding up to 10 minutes after application. Additional assessments include timing how quickly bleeding stops, rates of re-bleeding, and the need for additional applications or surgical intervention. Safety and efficacy will be observed through these measures and follow-up visits over the 28-day post-surgery period.

Researchers are evaluating emicizumab prophylaxis in people aged 1 month and older diagnosed with Type 3 von Willebrand disease (VWD). This Phase III, multicenter, open-label clinical study aims to assess the efficacy, safety, and how the body processes and responds to emicizumab. Participants previously receiving standard of care (SOC) on-demand therapy will be compared in a randomized manner, while those already on SOC prophylactic therapy will be evaluated based on prior study data. Participants are divided into three groups: those switching from on-demand SOC to emicizumab prophylaxis (Arm A), those continuing on-demand SOC therapy (Arm B), and those switching from SOC prophylaxis to emicizumab prophylaxis (Arm C). Emicizumab is given as weekly subcutaneous injections of 3 mg/kg for 4 weeks, followed by maintenance doses every two weeks. After 24 weeks, participants benefiting from emicizumab may continue it during an extension period, while those on SOC on-demand can switch to emicizumab in the extension. During the study, participants will undergo regular assessments measuring bleeding rates, safety events, and laboratory tests including blood levels of emicizumab and immune responses. Vital signs and questionnaires on pain and fatigue will be collected at scheduled intervals. Safety monitoring will continue up to nearly four years after the last dose, with detailed tracking of adverse events and heart and lung function. The study participation may last up to several years, including the treatment and extension phases.

Researchers are evaluating KLN-1010, a novel gene therapy, in patients with relapsed or refractory multiple myeloma to assess its safety, tolerability, and to determine the recommended Phase 2 dose. The trial is a Phase 1 study sponsored by Kelonia Therapeutics, Inc., focusing on patients who have previously undergone multiple treatments for their condition. Participants receive a single specified dose of KLN-1010, which is designed to generate anti-B Cell Maturation Antigen (anti-BCMA) CAR-T cells in the body. This gene therapy is administered once during the study, and its effects, including safety and pharmacokinetics, are closely monitored over time. During the study, participants undergo assessments for treatment-emergent adverse events and dose-limiting toxicities for up to 15 years after dosing. Pharmacokinetic evaluations of KLN-1010 and generated CAR-T cells occur up to two years post-infusion. Multiple myeloma status is monitored from dosing until disease progression or up to 15 years, with comprehensive safety and disease assessments throughout the follow-up period.

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 I/II 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 treatment for sepsis-induced coagulopathy, a condition where an infection causes an overactive immune response leading to harmful blood clots and low platelet levels. This condition can damage blood vessels and organs, sometimes resulting in death. The study aims to learn about the safety, suitable dose, and effects of the drug BAY 3389934 in patients receiving intensive care for sepsis-induced coagulopathy. Participants will receive BAY 3389934 as a continuous intravenous infusion over up to 96 hours. They will be divided into two groups: the first group receives a low starting dose which may be adjusted based on safety and tolerability, while the second group will receive a higher dose if the first group experiences no serious side effects. This phase 1 study focuses on carefully monitoring how the drug affects participants. During the approximately 28-day study period, doctors will collect blood and urine samples, perform physical exams, and monitor vital signs such as body temperature, blood pressure, and heart rate. Heart health will be assessed using electrocardiograms (ECG). Researchers will track any medical problems that occur during and after treatment to evaluate safety, including treatment-emergent adverse events and their severity. They will also measure blood clotting times from day 1 to day 6 to understand the drug’s effects.

Researchers are observing people with Type 3 von Willebrand disease (VWD) who are receiving preventive treatment as part of their regular care. This study aims to gather information on how well these treatments work and how safe they are, while also measuring participants' quality of life related to their health. The study is non-interventional, meaning it does not change the treatments participants receive but monitors outcomes over time. Participants aged 2 years and older who are already on standard preventive therapy for Type 3 VWD will continue their usual treatment as decided by their doctors. The study collects details about the doses and duration of Von Willebrand Factor concentrates, Factor VIII concentrates, recombinant activated Factor VII, and activated prothrombin complex concentrate used, following local treatment guidelines. Participants are expected to maintain their current treatment regimen throughout the observation period of at least 24 weeks. During the study, researchers will track bleeding rates, including treated and spontaneous bleeds, especially joint bleeds, and any adverse events according to World Health Organization standards. Health-related quality of life will also be assessed. Data collection will occur over a minimum of 24 weeks, allowing for comprehensive monitoring of treatment effectiveness and safety. Participants remain under their usual care, and no changes to their treatment are required by the study.

This research evaluates the use of oral anticoagulation (OAC) combined with background antiplatelet therapy in patients who develop new-onset post-operative atrial fibrillation (POAF) after isolated coronary artery bypass graft (CABG) surgery. The study aims to assess how effectively this approach prevents thromboembolic events and its safety regarding major bleeding. Patients who decline randomization may join a parallel registry to capture their treatment choices and risk profiles. Participants will be randomly assigned to one of two treatment groups: an experimental arm receiving OAC (either a vitamin K antagonist or a direct oral anticoagulant) along with antiplatelet therapy, or a control arm receiving antiplatelet therapy alone. The anticoagulation treatment lasts 90 days, and patients in the control group who experience recurrent atrial fibrillation after 30 days may switch to OAC. Follow-up includes clinic visits at 90 days and phone calls at 30, 60, and 180 days. A digital health substudy involving a wearable heart monitor for 30 days post-discharge is also offered to some participants. During the study, researchers will monitor outcomes such as death, stroke, transient ischemic attack, myocardial infarction, and thromboembolism up to 180 days after randomization, along with bleeding events at 90 days. Data collection includes medical record reviews, risk profiling, and use of anticoagulants and antiplatelets. Safety and effectiveness will be analyzed by comparing the two treatment strategies. Participation may last up to six months, with assessments conducted in person and by phone.