Bleeding Disorder

Researchers are evaluating the effects of NXT007 prophylaxis compared with emicizumab prophylaxis in people aged 12 years and older who have severe or moderate congenital hemophilia A without factor VIII (FVIII) inhibitors, or mild hemophilia A with FVIII inhibitors. The study aims to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of these treatments in managing hemophilia A. This is a Phase III, randomized, open-label clinical trial sponsored by Hoffmann-La Roche. Participants are randomly assigned to receive either NXT007 prophylaxis or emicizumab prophylaxis during the main study treatment period. NXT007 is given by subcutaneous injection using a combined drug-device product. Emicizumab is administered subcutaneously with a vial and syringe, starting with weekly loading doses of 3 mg/kg for 4 weeks, followed by maintenance doses at varying intervals and amounts depending on prior treatment. After the main treatment period, those on NXT007 may continue its use, and those on emicizumab can switch to NXT007 during an open-label extension phase. Participants will be monitored for at least 7 months during the main treatment period, with assessments focusing on the annualized bleed rate for treated bleeds. Additional evaluations include treatment burden, safety events such as adverse reactions and thromboembolic events, and measurement of drug levels and antibodies. Questionnaires will track the impact on daily activities and treatment satisfaction. The total study duration is approximately 3.5 years, with ongoing safety and efficacy follow-up throughout.

Researchers are evaluating the efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with Factor VIII (FVIII) prophylaxis in people aged 12 years and older with severe or moderate congenital hemophilia A without inhibitors. This Phase III randomized study aims to assess how NXT007, a newer treatment, compares to the current standard FVIII prophylaxis in preventing bleeding episodes in this population. Participants will be randomly assigned to receive either NXT007 prophylaxis, administered subcutaneously using a combined drug-device product, or standard FVIII prophylaxis given according to local guidelines. After the main treatment period of 6 months, those on NXT007 may continue with this dosing, while participants initially on FVIII can switch to NXT007 during an open-label extension phase. During the study, participants will be regularly monitored for bleeding rates, treatment burden, drug levels, and safety outcomes including adverse events and immune responses. Questionnaires will assess the impact of treatments on daily life and activities. The main measure is the annualized bleed rate over 6 months, with follow-up continuing for approximately 3.5 years to evaluate long-term effects and safety.

Researchers are conducting the GENESIS clinical study to map the HLA genomic region in the Greek population and explore its possible links with various underlying diseases. This non-interventional, multicenter study aims to provide a pilot map of genetic variation in HLA that may be useful in medical research and clinical applications related to selected diseases. The study plans to include 12,000 participants over a total duration of 36 months. Each participant will attend one visit at a participating site during which they will provide demographic data, lifestyle information such as smoking and alcohol use, blood pressure measurements, details on diagnosed diseases and treatments, and recent laboratory test results if available. Buccal swab samples will be collected from each participant to extract DNA for HLA genotyping analysis. Selected samples will undergo further whole genome sequencing to investigate associations with autoimmune diseases. Participants will receive a personalized ancestry report after analysis completion. During the study visit, data collection includes demographic and health information, as well as laboratory and clinical test results from the past year. The genetic material from buccal swabs will be stored and processed for genetic analysis. Researchers will measure allele frequency of HLA alleles in the Greek population and assess the prevalence and risk associations of selected HLA-related diseases. The study's total duration is 36 months with results available at the end of this period.

This research aims to learn about the safety and effects of marstacimab, a study medicine being evaluated for treating Hemophilia in pediatric patients. It focuses on children aged 1 to 17 years with severe Hemophilia A or moderate to severe Hemophilia B, including those with or without inhibitors. The study compares participant experiences on marstacimab with their past standard treatments to see if it helps prevent bleeding episodes common in Hemophilia. All participants will receive weekly marstacimab injections under the skin. The first dose is administered at the study site, and subsequent doses during the 12-month treatment period can be given at home or the study site. The study enrolls participants in age groups sequentially, starting with adolescents (12-17 years), followed by children 6-11 years, and then 1-5 years. The full participation lasts about 14 months, including screening, treatment, and follow-up periods. Participants will visit the study site at least 10 times and have 6 scheduled phone calls every two months. Researchers will monitor bleeding rates, adverse events, immune reactions, and joint health, along with quality of life changes. Safety assessments cover the entire study period, including follow-up, to evaluate marstacimab's effects and tolerability in children with Hemophilia.

Primary immune thrombocytopenia (ITP) is a condition where the immune system mistakenly destroys platelets, leading to a lower platelet count and increased risk of bruising or bleeding. This research aims to evaluate the long-term safety, tolerability, effectiveness, and how the body processes mezagitamab in adults with chronic primary ITP. Participants who previously took part in related mezagitamab studies (TAK-079-3002 and TAK-079-1004) are invited to join this continuation study. Eligible participants will receive mezagitamab injections under pre-specified criteria and as needed, based on their clinical condition and the investigator's judgment. This on-demand treatment may be repeated multiple times during the study. The study is an open-label, phase 3 trial that monitors participants over an extended period with repeated dosing as required. Participants will visit the study clinic multiple times for assessments throughout the study, which may last up to approximately 108 weeks. Researchers will monitor treatment-emergent adverse events, platelet response duration, use of other ITP medications, and the presence of antibodies against the drug. Blood samples will be collected to measure drug levels and immune responses. Safety and effectiveness will be closely followed, including any need for rescue therapies.

Researchers are evaluating the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A, a condition characterized by low levels of factor VIII. This phase 1 and 2 study is sponsored by Hoffmann-La Roche and focuses on the effects of this gene therapy treatment in a specific patient group. The trial aims to understand the side effects and safety profile of SPK-8011QQ over an extended period. Participants will receive a single intravenous infusion of SPK-8011QQ on the first day of the study. The treatment involves delivering the gene therapy directly into the bloodstream. The study is open-label and conducted across multiple centers, with all participants receiving the experimental treatment. No placebo or comparison groups are involved in this single-arm trial. During the study, participants will be followed for up to approximately five years to monitor adverse events, including their incidence, severity, and any serious or treatment-related occurrences. Laboratory tests will be conducted regularly to detect any abnormal values. Participants will also complete questionnaires and attend scheduled visits to assess their health and response to the therapy throughout the study duration.

Researchers are studying the use of unlicensed cryopreserved cord blood units (CBUs) for transplantation in both pediatric and adult patients with various blood-related cancers and other disorders affecting the blood-forming system. This observational study aims to evaluate outcomes such as the recovery of a certain level of white blood cells after transplantation, as well as the incidence of infections, infusion reactions, survival rates, and graft-versus-host disease over time. The study involves patients receiving unlicensed CBUs at multiple U.S. transplant centers. These CBUs are used for patients with hematologic malignancies and other blood disorders. The protocol collects data on patients who receive these unlicensed transplant units, without administering a new treatment but observing the outcomes after transplantation. Participants will be monitored for neutrophil recovery at 60 and 100 days post-transplant, along with assessments of infection transmission, infusion reactions, survival one year after transplant, and occurrences of acute and chronic graft-versus-host disease. Platelet engraftment levels will also be tracked. The study includes patients of any age and follows them through the transplantation and recovery process to gather information on these key outcomes.

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 how different doses of a study medicine called Inno8 work in the bodies of people with haemophilia A. This study aims to see if Inno8 is safe for use in people with this condition. The study medicine is new and not yet available by prescription. The study will last about 11 weeks. Participants will receive oral doses of the study drug NNC0442-0344 A in three different groups, each getting a dose to test how the medicine acts in the body. All doses are given by mouth, and the study includes multiple ascending doses to monitor safety and how the drug is processed. During the study, participants will be closely monitored for any side effects or treatment emergent adverse events from the first day of dosing through day 46. Researchers will measure blood markers related to coagulation and immune response, such as D-dimer, prothrombin fragments, fibrinogen, platelets, and anti-Inno8 antibodies. They will also assess drug concentration levels and thrombin generation to understand the drug's effects. The total participation lasts about 11 weeks, including follow-up.

Researchers are evaluating a new medicine called Inno8 to understand how eating and drinking before and after taking the medicine affects its absorption in the stomach. The study focuses on healthy male participants aged 18 to 45 years and aims to learn how meal timing influences the way Inno8 behaves in the body. This is a Phase 1, randomized, open-label study sponsored by Novo Nordisk A/S. Participants will take a single oral dose of Inno8 after fasting overnight, with fasting duration varying depending on the group they are assigned to. The study includes four groups, with each receiving the same oral dose of the medicine but under different meal timing conditions. After taking the dose, participants will fast again as specified by their group. The entire study lasts up to 9.5 weeks. During the study, participants will undergo assessments including blood tests to measure Inno8 concentration over time and monitor safety markers such as clotting factors and adverse events. Researchers will measure how much medicine is absorbed and the timing of peak levels in the blood. Safety evaluations, including laboratory tests and vital signs, will be conducted from the first dose up to 36 days. Participants' health will be closely monitored throughout the study period.