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 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 evaluating a new tool to predict the risk of postpartum hemorrhage PPH, a serious complication during and after childbirth. The study compares the current basic risk assessment method with an advanced model that uses 21 factors to calculate a more precise risk. This model updates automatically during labor and provides clinicians with a Best Practice Advisory BPA suggesting recommended actions when risk is elevated. Participants will receive standard care, including the usual category-based risk assessment. Those in the intervention group will also have the new risk prediction displayed, which triggers the BPA if the risk is 3% or higher. This advisory includes recommendations such as preventative use of tranexamic acid and second-line uterotonics, guiding providers without requiring mandatory actions. The study aims to see if this enhanced approach improves outcomes for mothers during childbirth. Throughout the study, researchers will collect data on postpartum morbidity and mortality from delivery until hospital discharge and up to 30 days after birth. They will monitor estimated blood loss, treatments for hemorrhage, blood transfusions, complications, and fetal APGAR scores at 1, 5, and 10 minutes. The total participation duration typically covers delivery through 30 days postpartum, focusing on the safety and health of both mother and baby.

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 systems 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 mgkg 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.

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 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 patients routine care and treatment schedule.

This research aims to evaluate the effects of TAK-330 compared to standard four-factor prothrombin complex concentrate 4F-PCC in adults treated with Factor Xa inhibitors who need urgent surgery or invasive procedures. The study focuses on reversing anticoagulation caused by Factor Xa inhibitors and is a Phase 3, randomized, triple-blind trial sponsored by Takeda. Participants will be randomly assigned to receive either TAK-330 or the standard 4F-PCC. TAK-330 is given as a single intravenous infusion dose of 25 IUkg before surgery, with an optional additional 25 IUkg dose during surgery if needed, not exceeding a total of 50 IUkg or 5,000 IU. The standard 4F-PCC dose and infusion speed follow local protocols, with an additional dose allowed during surgery under the same maximum limits. These treatments are administered during hospitalization prior to surgery. During the study, participants will be hospitalized for the surgery and treatment administration, with assessments of hemostasis effectiveness at surgery end and 24 hours after treatment. Researchers will monitor blood product use, adverse events including thrombotic events, and participant survival up to 30 days after surgery. Follow-up will also include contact via telehealth or phone 30 days post-surgery to evaluate safety and outcomes. Total participation duration covers the hospital stay and the 30-day post-surgery monitoring period.

Researchers are evaluating the effectiveness and safety of two hemostatic products, TachoSil and Surgicel Original, as aids to control mild to moderate soft tissue bleeding during surgery. This study focuses on patients undergoing elective open abdominal, retroperitoneal, pelvic, or thoracic surgeries, including certain transplant surgeries except liver or heart transplants. The trial aims to compare these two products in managing bleeding at targeted bleeding sites TBS with mild to moderate severity. Participants will be randomly assigned to receive either TachoSil or Surgicel Original applied as an adjunct to control bleeding during surgery. Both are biological products used to help stop bleeding. The intervention involves applying and firmly pressing the product at the bleeding site for up to 3 minutes after randomization. The study will monitor hemostatic success within 3 and 6 minutes after application, as well as intervention failures. During the study, participants will be assessed for bleeding control effectiveness and safety. Researchers will record bleeding status using the VIBe Scale and monitor vital signs, hemoglobin levels, platelet counts, and coagulation parameters. The primary outcome is hemostatic success within 3 minutes of treatment. Participants must meet eligibility criteria including age from 1 month and older and specific surgical and bleeding characteristics. The study is expected to continue until December 31, 2026.

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.