Hemophilia

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.

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.

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 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.

Researchers are evaluating the safety and effectiveness of the ETHIZIA patch compared to SURGICEL Original for controlling minimal, mild, or moderate soft tissue bleeding during open surgeries. The study focuses on surgeries involving the abdomen, retroperitoneal area, pelvis, thoracic region (excluding heart surgery), and extremities. The goal is to see which device better achieves bleeding control within 3 minutes after application and prevents re-bleeding up to 10 minutes later. Participants will be randomly assigned to receive either the ETHIZIA patch or SURGICEL Original applied directly to bleeding soft tissue sites during surgery. Both treatments are applied intraoperatively at bleeding sites where conventional methods like sutures or cautery are not effective or practical. After surgery, participants will be followed for 28 days to monitor outcomes and any potential complications. During the study, researchers will closely monitor bleeding control at the target site, measuring the percentage of cases achieving hemostasis at 3 minutes and the absence of re-bleeding up to 10 minutes after application. Additional assessments include timing how quickly bleeding stops, rates of re-bleeding, and the need for additional applications or surgical intervention. Safety and efficacy will be observed through these measures and follow-up visits over the 28-day post-surgery period.

Researchers are evaluating the safety and tolerability of BBM 002 injection, a gene therapy using an adeno-associated virus (AAV) vector that carries the human factor VIII gene. This study focuses on males with Hemophilia A who have very low levels of factor VIII (less than or equal to 2 IU/dl). The trial is an early phase 1, single-arm, open-label study designed to assess this investigational genetic treatment. Participants will receive a single intravenous dose of BBM 002 at 1×10^13 vector genomes per kilogram. BBM 002 is designed to increase the body's production of factor VIII, which is important for blood clotting. This one-time infusion is the only treatment administration in the study. During the study, participants will be monitored for safety, including dose limiting toxicities within 10 weeks and treatment-emergent and serious adverse events over 52 weeks. Researchers will evaluate participants' bleeding history, factor VIII levels, and adverse reactions. The total follow-up duration is at least one year to track safety and response to the gene therapy.

Researchers are studying the drug SR604 in both healthy adults and individuals with Hemophilia A, Hemophilia B, or Factor VII deficiency. The study aims to evaluate the safety, tolerability, how the drug moves and works in the body, and its effectiveness in treating these bleeding disorders. This first-in-human trial includes a phase for healthy participants as well as a phase for affected participants, with both groups monitored closely for any adverse events or changes in blood clotting markers. In the first part, healthy participants receive a single subcutaneous injection of SR604 or a placebo in ascending doses across multiple groups. In the second part, participants with the bleeding disorders receive multiple subcutaneous injections of SR604 every four weeks at increasing dose levels. The study uses a randomized and double-blind design to compare SR604 with placebo for healthy participants, while affected participants receive only SR604. The total study participation lasts about three months. During the study, participants undergo medical examinations, blood tests, and monitoring for treatment-related side effects and changes in blood coagulation. Researchers assess drug levels in the blood at various times and track bleeding events in participants with bleeding disorders. Safety is closely monitored through adverse event reporting and antibody testing. Participants also provide medical history and may undergo a treatment weaning period before starting SR604. The study measures responses up to three months from the first dose to understand the drug's effects and safety profile.

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.