Bleeding Disorder

Researchers are evaluating the safety, effectiveness, and how the body processes and responds to 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 any severity of hemophilia A with FVIII inhibitors. This phase 3, randomized, open-label study aims to compare these treatments to better understand their impact on bleeding rates and treatment burden. Participants will be randomly assigned to one of two main treatment groups. One group will receive NXT007 prophylaxis administered subcutaneously using an integrated drug-device combination product. The other group will receive emicizumab prophylaxis via subcutaneous injections, starting with weekly loading doses for 4 weeks, then maintenance dosing at various intervals depending on prior treatment status. After the main treatment period, participants from both arms can continue or switch to NXT007 in an open-label extension phase. Throughout the study, participants will be closely monitored with regular assessments, including measuring annualized bleed rates for different types of bleeds, treatment burden questionnaires, and safety evaluations such as adverse event monitoring and laboratory tests. These evaluations will continue throughout approximately 3.5 years of study participation to provide comprehensive data on treatment effects and safety.

Researchers are evaluating the effectiveness, safety, and how the body processes and responds to NXT007 prophylaxis compared to Factor VIII FVIII prophylaxis in people aged 12 years and older with severe or moderate congenital hemophilia A who do not have inhibitors. This phase III study focuses on participants who have previously been treated with FVIII prophylaxis. The goal is to understand how NXT007 performs against the current standard treatment for this condition. Participants will be randomly assigned to receive either NXT007 prophylaxis, given as a subcutaneous injection with an integrated drug-device combination product, or standard Factor VIII prophylaxis according to local dosing and frequency guidelines. After the main six-month treatment period, those receiving NXT007 may continue this treatment in an open-label extension, and those initially on FVIII prophylaxis may switch to NXT007 during this extension phase. During the study, participants will be closely monitored through various assessments, including tracking the annualized bleed rate ABR for treated bleeds over six months, questionnaires evaluating treatment burden and impact on social and recreational activities, and safety evaluations such as adverse events, injection-site reactions, and antibody development against NXT007. The study will continue follow-up for approximately 3.5 years to gather comprehensive data on treatment effects and safety.

The GENESIS clinical study aims to map HLA genetic variation in the Greek population and evaluate possible correlations with selected underlying diseases. It is a multicenter, prospective, non-interventional clinical study targeting 12,000 subjects over an anticipated duration of 36 months, with the goal of creating a pilot HLA map for medical research and possible clinical applications. Each subject will complete one visit at a participating site and provide demographic information, including date of birth, gender, race, ancestry, height, and weight, as well as information about smoking or vaping, alcohol consumption, arterial blood pressure, diagnosed diseases, and current treatments. Recent clinical laboratory results from up to 12 months before sample collection may also be collected when available, including blood count, metabolic, liver enzyme, and biochemical parameters. Two buccal swabs will be collected from each subject for DNA extraction and HLA genotyping analysis. Selected DNA samples will also undergo low-pass whole genome sequencing to further investigate associations between the HLA region and autoimmune diseases. After the analysis is completed, an individualized ancestry report will be securely available to study subjects if they elect to access it.

Researchers are evaluating the safety and effects of marstacimab, a study medicine, for potential treatment of hemophilia in boys and male children aged 1 to 17 years. The trial focuses on those with severe Hemophilia A or moderately severe to severe Hemophilia B, including participants with or without inhibitors. The study aims to compare participants experiences with marstacimab to their historical bleeding episodes without the medicine. All participants will receive weekly subcutaneous injections of marstacimab. The first dose is administered at the study site by staff, and subsequent doses can be given at home or by study staff during the 12-month treatment period. Enrollment opens sequentially by age groups, starting with adolescents 12-17 years, followed by children 6-11 years, and finally children 1-5 years. The study includes about 1 month of screening, 12 months of treatment, and 1 month of follow-up. Participants will visit the study site at least 10 times during the study, with the option for two visits to occur at home if allowed. They will also receive six phone calls approximately every two months. Researchers will monitor bleeding rates, adverse events, injection site reactions, and immune responses. The overall participation lasts about 14 months, including screening, treatment, and follow-up periods.

Primary immune thrombocytopenia ITP is a condition where the immune system mistakenly destroys platelets, leading to a lower number of platelets and increased risk of bruising or bleeding. This Phase 3 study evaluates the long-term safety, tolerability, and effectiveness of mezagitamab in adults with chronic primary ITP. The study also investigates how the body processes mezagitamab over an extended period. Participants who completed previous mezagitamab studies TAK-079-3002 or TAK-079-1004 will be invited to join this continuation trial. Eligible participants may receive mezagitamab injections on demand, with treatment courses repeated as needed based on specific criteria and the investigators clinical judgment. The treatment is administered subcutaneously. During the study, participants will visit the clinic several times for assessments. Researchers will monitor safety by tracking treatment-emergent adverse events, and evaluate effectiveness through platelet response and remission rates. Measurements of drug levels and antibodies will also be taken. The study may last up to approximately 108 weeks, allowing detailed long-term follow-up.

Researchers are evaluating the safety and tolerability of SPK-8011QQ, a gene therapy, in adult males with moderately severe to severe hemophilia A. This study is a Phase 2b, single-arm, open-label trial focusing on individuals with low factor VIII activity levels who have a history of treatment with factor VIII products. The purpose is to carefully monitor adverse events and treatment effects over an extended period. Participants will receive a single intravenous infusion of SPK-8011QQ on the first day of the study. The study is designed as a single group treatment with no placebo comparator. After the infusion, participants will be followed for up to approximately five years to assess safety and tolerability outcomes, including adverse events and laboratory abnormalities. Throughout the study, participants will attend scheduled visits where safety assessments will be conducted. These include monitoring for adverse events categorized by severity and type, laboratory tests, and antibody screenings. The primary outcomes focus on the frequency and severity of adverse events related to the treatment. The study duration may extend up to five years, allowing for long-term safety monitoring in participants.

Researchers are studying the use of unlicensed cryopreserved cord blood units CBUs for transplantation in both children and adults with blood cancers and other related disorders. This observational study involves patients with hematologic malignancies and various inherited and acquired disorders affecting the blood and immune system. The main goal is to monitor how well neutrophil recovery occurs after transplantation using these unlicensed CBUs in multiple institutions. Participants receive unlicensed cryopreserved CBUs as part of their transplant treatment. The study includes patients of any age receiving these CBUs for approved indications. The protocol focuses on the access and distribution of these unlicensed units rather than a specific treatment intervention. The study gathers data from recipients who receive these CBUs, tracking outcomes after transplantation. Participants are monitored for neutrophil recovery at 60 and 100 days after transplant, defined by a neutrophil count of at least 500mm3. Researchers also collect information on infection transmission, infusion reactions, survival rates at one year, and incidence of acute and chronic graft versus host disease. Platelet recovery is also evaluated. Safety and efficacy outcomes are followed over time to better understand the effects of unlicensed CBUs in this patient population.

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 oral medicine called Inno8 in people with haemophilia A, a condition characterized by low factor VIII activity. The study aims to understand how different doses of Inno8 are processed in the body and to assess its safety. This clinical trial is a Phase 1 study sponsored by Novo Nordisk AS and will last about 11 weeks. Participants will receive oral doses of the study drug NNC0442-0344 A in one of three experimental cohorts. The trial is non-randomized and open-label, meaning all participants receive the study drug without placebo or blinding. The dosing occurs over several days, and the study includes follow-up assessments to monitor drug levels and effects. During the study, participants will have regular assessments to monitor safety and how the drug behaves in the body. Measurements include tracking treatment-emergent adverse events, laboratory tests for blood clotting factors, antibody development, and drug concentration levels at specified times. The total participation duration is approximately 11 weeks, including dosing and follow-up to ensure safety and gather detailed pharmacokinetic and pharmacodynamic data.

Researchers are evaluating Mim8, a new medicine designed to help people with haemophilia A, including those with or without inhibitors. Mim8 aims to prevent bleeding episodes by replacing the function of the missing clotting factor VIII. This long-term study will last up to 5.5 years, ending either when Mim8 is approved in the participants country or by June 2028, whichever comes first. The study includes participants who have been involved in earlier related studies or are new infants with severe haemophilia A. Participants will receive Mim8 as a preventive treatment through subcutaneous injections. Depending on their entry point, participants may use an enhanced cartridge or a DV3407 pen-injector device for administering Mim8. The treatment is given regularly over the study period, with participants potentially receiving up to 262 injections. In the event of bleeding, additional haemostatic medications may be used as agreed with the study doctor. Female participants who are pregnant, breastfeeding, or planning pregnancy during the study are not eligible. During the study, participants will be monitored for any side effects, including injection site reactions and the development of antibodies against Mim8. Researchers will also track bleeding episodes, Mim8 blood levels, and device handling for some participants. Participants and their representatives will complete diaries and questionnaires about their treatment and health. Safety will be carefully followed throughout the study, which may last several years depending on individual enrollment and study progress.