Acquired Hemophilia

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 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 the safety of damoctocog alfa pegol, a medicine used to treat hemophilia A, a genetic bleeding disorder caused by missing clotting factor 8 (FVIII). This study focuses on Korean participants who are at least 12 years old and have previously used other hemophilia A treatments. The main goal is to learn more about the safety of this medicine when used in routine medical care. Participants receive damoctocog alfa pegol as prescribed by their doctors following usual clinical practices. The study does not provide the medicine directly, and no changes to healthcare or additional treatments are made for study purposes. Data collection happens continuously during a 36-week period as participants continue their regular clinic visits. During the study, researchers collect data from participants' health records and routine care without requiring extra visits or tests. They monitor for any medical problems, including adverse events, serious adverse events, and reactions related to the treatment. The study tracks bleeding episodes, medicine usage, and treatment choices over about eight months for each participant, providing real-world safety information.

Researchers are conducting a survey in Japan to study the use of Susoctocog Alfa (Genetical Recombination) intravenous injection in participants with bleeding events caused by acquired Hemophilia A (AHA). The study aims to monitor side effects related to this treatment and assess its impact on bleeding events in those affected by AHA. The sponsor provides guidelines for clinic data recording but does not influence the treatment methods. Participants will receive Susoctocog Alfa (Genetical Recombination) through intravenous injection following their clinic's usual care practices. The study does not alter how the treatment is given but observes its use in real-world settings. Participants will be followed for side effects and treatment effects during their care. During the study, doctors will monitor participants for any treatment-related side effects up to 90 days after the last dose or until the participant stops treatment. Researchers will collect data on adverse events and evaluate the effectiveness of the treatment on bleeding episodes. The total duration of monitoring varies by participant, based on their treatment timeline and study discontinuation status.

This research focuses on boys with Hemophilia A who have developed inhibitors against factor VIII (FVIII) treatment. Hemophilia A is a genetic bleeding disorder caused by deficient FVIII, leading to frequent bleeding episodes that can cause joint damage and long-term health problems. Inhibitors are antibodies that neutralize FVIII and make standard replacement therapy ineffective, representing a major treatment challenge. The study aims to evaluate the safety and effectiveness of various dosing regimens of immune tolerance induction (ITI) therapy using FVIII to eradicate these inhibitors and improve patient outcomes. Participants receive different doses and frequencies of ADVATE4, a recombinant FVIII product, to induce immune tolerance. The study includes multiple groups based on inhibitor levels and treatment history: low inhibitor titer groups receive either non-daily or daily doses of 50 or 100 IU/kg; high inhibitor titer groups receive low, medium, or high doses of ADVATE, some combined with immunosuppressive agents for those with poor prognosis. This multicenter, open-label trial is designed to explore the impact of dose and frequency on ITI success and to examine related biomarkers. During the study, boys up to 18 years old with severe Hemophilia A and inhibitors will be closely monitored over 24 months to assess treatment success. Researchers will track inhibitor levels, immune markers, bleeding episodes, and safety outcomes. Patients undergo regular infusions of ADVATE according to assigned dosing schedules, with clinical assessments and laboratory tests to evaluate immune tolerance induction. The study also aims to identify predictors of ITI outcomes and relapse risks to improve personalized treatment strategies for children with Hemophilia A and inhibitors.

Researchers are evaluating whether artificial intelligence (AI) models can accurately interpret ROTEM (Rotational Thromboelastometry) data and make treatment recommendations for adult patients undergoing elective cardiac or liver transplant surgery. This observational validation study aims to compare AI models' decisions to those of expert clinicians to see if AI can match expert-level interpretation in detecting coagulopathy types and suggesting treatments. The study uses de-identified ROTEM data collected during surgery, which is converted into clinical vignettes for review. AI models such as ChatGPT and Gemini analyze these standardized ROTEM parameters and recommend treatments following established ROTEM-guided transfusion protocols. Their outputs are compared to a panel of three clinical experts, forming the gold standard for diagnosis and treatment decisions. Participants will undergo standard ROTEM testing during their elective surgery. Researchers will evaluate agreement between AI and expert panels on whether treatment is needed and on the type of coagulopathy present. The study involves answering 14 structured clinical questions for each case, with results measured by accuracy and concordance metrics. No patient treatment is changed based on AI results, and all data is anonymized with informed consent obtained. The study timeline extends through December 2025.

Researchers are studying people with various non-cancerous blood disorders to better understand the long-term safety and effectiveness of treatments used for these conditions. This observational study aims to collect consistent and high-quality data over time from patients with congenital or acquired hematologic and connective tissue disorders that cause bleeding or clotting problems. It addresses the need for reliable long-term information beyond initial drug approval trials and includes multiple disease-specific groups for comprehensive research. Participants will join one of several cohorts based on their specific blood disorder, such as hemophilia, von Willebrand disease, congenital platelet disorders, rare bleeding disorders, thrombosis/thrombophilia, or other non-neoplastic hematologic conditions. The study includes several arms and modules focusing on different therapies or patient groups, like previously untreated patients, gene therapy outcomes, and treatment with specific drugs (e.g., ALTUVIIIO® or Hemlibra®). Data collection occurs at enrollment and regularly every six months and annually, with additional data gathered as needed. During the study, participants will undergo various assessments including medical history, treatment records, bleeding event tracking, and biospecimen collection for future research. The study will follow participants for at least 15 years to monitor therapy safety and effectiveness. Researchers will analyze dosing patterns, bleeding rates, and real-world treatment outcomes while maintaining long-term participant follow-up and data quality.