Hemorrhage Disorder

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 studying postpartum hemorrhage (PPH), a serious complication during and after childbirth that can cause significant health problems for mothers. This trial aims to improve how the risk of PPH is predicted using a new computer model that considers 21 factors and updates automatically during labor. The study compares this new model combined with a provider-facing alert system to current basic risk tools, with the hope of improving outcomes for mothers. The study includes two groups: one receives the usual care with a traditional category-based risk assessment, and the other group receives standard care plus the new risk prediction model. If the new model predicts a risk of 3% or higher, it triggers an alert recommending best practice steps like preventive medications. Providers can decide whether to follow these recommendations. Both groups receive standard care risk assessments as part of their admission process. Participants are all women admitted for vaginal or cesarean delivery at the medical center. Researchers will collect and compare data on a range of health outcomes related to bleeding and complications from the time of delivery until 30 days later. They will use hospital records to measure blood loss, treatments given, length of stay, and other health events. The study aims to see if the new model and alerts lead to better health outcomes for mothers during and after childbirth.

Researchers are evaluating high-dose cyclophosphamide as an induction therapy for adult secondary hemophagocytic lymphohistiocytosis (HLH), a severe condition involving excessive immune cell activation that can quickly lead to organ damage and early death within 30 days. Secondary HLH arises from tumors, autoimmune diseases, infections, and other causes, and current treatments have high early mortality rates. This study aims to find a more effective induction treatment for non-infective adult HLH by targeting the immune system's overactive T cells. Participants will be randomly assigned to receive either the experimental treatment with high-dose cyclophosphamide or the standard HLH2004 treatment regimen. The cyclophosphamide group will receive 40 mg/kg intravenously once daily for two days. The standard treatment includes drugs such as etoposide, dexamethasone, and cyclosporine administered over several weeks with specified dosing schedules. This design allows comparison of efficacy and safety between the new high-dose therapy and established treatment. During the 30-day study period, participants will be monitored for response to treatment, including complete or near-complete remission rates, partial responses, and mortality. Key measures include fever reduction time and improvements in diagnostic criteria indicators. Safety and treatment effectiveness will be assessed through clinical observations and laboratory tests. Participants will be followed closely to evaluate outcomes and any adverse effects related to the therapies.

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 studying adults with Acquired Hemophilia A (AHA), a rare bleeding disorder where blood does not clot properly, leading to sudden and severe bleeding. This study aims to evaluate the safety of OBIZUR injection in adults with AHA and to observe how the treatment controls bleeding in real-world clinical settings. The study collects both past and ongoing data from participants receiving OBIZUR as part of their routine care. This is a single-arm, observational study involving participants who are either currently receiving OBIZUR or are starting OBIZUR treatment for the first time. Data will be collected retrospectively from the first OBIZUR treatment after approval in Korea and prospectively from the time participants consent to join the study. Treatment decisions and follow-up care are managed by the treating physicians according to standard practice. Participants will have their existing medical records reviewed, and new health information will be collected during the study. Researchers will monitor adverse events, bleeding control effectiveness, and treatment usage over approximately three months. The study focuses on safety outcomes such as adverse events and drug reactions, as well as measures of bleeding control like time and dose needed to stop bleeding. The total participation time varies based on each patient's routine care and treatment schedule.

Researchers are evaluating the effects of TAK-330 compared with a standard treatment known as four-factor prothrombin complex concentrate (4F-PCC) for reversing anticoagulation caused by Factor Xa inhibitors in adults needing urgent surgery or invasive procedures. This Phase 3 trial aims to determine which treatment better controls bleeding during and after surgery in patients who require rapid reversal of blood thinning effects. Participants will be randomly assigned to receive either TAK-330 or the standard 4F-PCC treatment before surgery. TAK-330 is given as a single intravenous infusion of 25 international units per kilogram on the day of surgery, with an optional additional dose during surgery if needed, not exceeding a total dose of 50 IU/kg or 5,000 IU. The standard 4F-PCC is administered according to local protocols, with a possible additional dose during surgery under similar dosing limits. During the study, patients will be hospitalized and monitored closely. Researchers will assess bleeding control during surgery, blood product use, and adverse events up to 30 days after surgery. Follow-up contact will be conducted by phone or telehealth 30 days post-surgery. The main outcome measured is the percentage of participants achieving effective bleeding control at the end of surgery.

Researchers are evaluating the safety and effectiveness of TachoSil compared to Surgicel Original, a local hemostatic product used for over 60 years, to control mild to moderate soft tissue bleeding during surgery. This Phase 3 clinical trial focuses on patients undergoing elective open abdominal, retroperitoneal, pelvic, or thoracic surgeries, excluding certain types like liver or heart transplants. The goal is to determine which product better helps stop bleeding within minutes during surgery. Participants will be randomly assigned to receive either TachoSil or Surgicel Original applied directly to the bleeding site as an adjunct to standard surgical methods. The study assesses hemostatic success within 3 minutes as the primary measure, with additional evaluations at 6 minutes. The intervention involves pressing the study product firmly on the targeted bleeding soft tissue site until 3 minutes post-application. Surgeries involving cardiovascular, hepatic, laparoscopic, and robotic approaches are excluded. During the trial, participants will be closely monitored intra-operatively for bleeding control using the VIBe Scale to grade bleeding severity. Researchers will collect data on success rates of hemostasis, intervention failures, and safety outcomes. Screening includes blood tests to ensure adequate hemoglobin, platelets, and coagulation status. The study runs from April 2025 to June 2026, with participants followed during surgery to assess immediate treatment effects and safety.

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 ALN-6400 in a combined Phase 1/2 clinical trial involving healthy adult volunteers and adult patients with Hereditary Hemorrhagic Telangiectasia (HHT). The study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of single ascending doses of ALN-6400 in healthy volunteers and multiple doses in patients with HHT. This investigation also explores the efficacy of ALN-6400 in managing symptoms related to HHT. The study is divided into two parts: Part A includes healthy volunteers receiving a single dose of ALN-6400 or a placebo, and Part B involves patients with HHT receiving multiple doses of ALN-6400 or placebo. Both treatments are administered subcutaneously. Participants are randomly assigned to either the experimental or placebo groups, and the study is triple-blinded to ensure unbiased results. Participants will be monitored for safety through the frequency of adverse events up to 36 weeks in healthy volunteers and up to 96 weeks in patients with HHT. Researchers will measure drug concentrations in plasma, changes in plasminogen protein and activity levels, and various clinical outcomes related to epistaxis severity and frequency, hematologic support, and quality of life using patient-reported outcomes. The study includes long-term follow-up assessments to track these parameters throughout the study 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.