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 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 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.

Researchers are observing people with hemophilia A, a genetic bleeding disorder caused by a lack of clotting factor 8 FVIII, who use the drug damoctocog alfa pegol as prescribed by their doctors. The study aims to learn more about the safety of this drug in Korean participants who have previously used other hemophilia A treatments. Hemophilia A can cause excessive bleeding after injury or surgery, and damoctocog alfa pegol replaces the missing FVIII to help prevent or treat bleeding episodes. Participants will continue their usual treatment with damoctocog alfa pegol during routine medical care, following approved product guidelines. No study drug is provided by the researchers, and no changes to healthcare are made for study purposes. Data collection occurs over about 8 months for each participant, from December 2023 to March 2026, with information gathered from health records and routine clinic visits. Participants are involved through their regular medical visits, with no extra tests or visits required for the study. Researchers will monitor and collect data on any medical problems, including various types of adverse events and reactions, during treatment. They will also track bleeding episodes and treatment use, comparing with previous FVIII products. Safety data will be collected continuously during the 36-week observational period without interfering with standard care.

This research aims to evaluate the use of Susoctocog Alfa Genetical Recombination intravenous injection in participants with bleeding events caused by acquired Hemophilia A AHA in Japan. The study focuses on monitoring side effects related to this drug and assessing its impact on bleeding episodes in this condition. The sponsor will not influence treatment decisions but will guide clinics on how to document patient outcomes. Participants will receive Susoctocog Alfa Genetical Recombination intravenously according to their clinics standard practice for AHA. The study observes the treatment as it is normally given without altering care. There is no placebo or comparison group. The drug administration and monitoring are part of routine clinical care during the study period. Throughout the study, participants will be followed for side effects from the first dose until up to 90 days after the last dose or until they stop treatment. Researchers will collect data on adverse events and treatment effectiveness as judged by investigators. The total study duration per participant varies depending on treatment and discontinuation timing, with safety checks continuing after treatment ends.

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 IUkg 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 conducting a long-term observational study called ATHN Transcends to better understand non-cancerous blood disorders. This study follows participants with various hematologic conditions, including hemophilia, von Willebrand Disease, congenital platelet disorders, rare bleeding disorders, thrombosis, and other related conditions. The aim is to collect uniform and high-quality data on the safety, effectiveness, and treatment practices of new and existing therapies over many years, addressing gaps beyond initial clinical trials and real-world treatment outcomes. Participants are assigned to specific cohorts based on their diagnosis, such as Hemophilia, Congenital Platelet Disorders, and others. Within these cohorts, there are multiple study arms and modules focusing on different therapies or disease aspects, including gene therapy outcomes and product-specific data collection. Data is gathered through scheduled assessments at enrollment, every six months, annually, and as needed, with follow-up planned for a minimum of 15 years. The study also includes a biorepository for collecting biological samples to support current and future research. Throughout the study, participants provide information during regular visits, and data is collected about their medication use, bleeding events, and treatment effectiveness. Researchers monitor safety and treatment outcomes continuously to better understand long-term effects. The study involves comprehensive evaluations but does not involve administering treatments instead, it observes participants receiving usual care. Participants may remain in the study for many years, contributing to a valuable resource that helps improve understanding of blood disorders and their management.

Researchers are gathering health information from people with blood disorders to better understand their quality of life and health outcomes. This observational study collects detailed data to help doctors, scientists, and policymakers find better ways to treat blood disorders like hemophilia, thrombosis, sickle cell disease, and others. The study is supported by the American Thrombosis and Hemostasis Network ATHN and aims to answer important scientific and public health questions. Participants health data will be collected and regularly updated in a secure registry called the ATHNdataset. Information includes demographics, diagnosis details, family history, physical exams, vital signs, lab and genetic tests, imaging results, medications, treatments, surgeries, immunizations, and patient-reported outcomes. Data is gathered from routine care visits at ATHN Affiliate centers and may be used for research, advocacy, safety monitoring, and quality improvement projects. Participants will share their health information through encounters with ATHN Affiliate care providers. The study team will analyze this comprehensive data over 15 to 20 years to support clinical care and public health reporting for the blood disorders community. All relevant health details, questionnaires, and treatment information are securely stored and used to improve understanding and management of blood disorders.