Hemophilia

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.

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.

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.

Diffuse large B-cell lymphoma DLBCL is the most common and aggressive subtype of non-Hodgkin lymphoma, with rapid tumor growth and symptoms varying by tumor location. The disease is classified using advanced methods like immunohistochemistry and molecular testing to guide prognosis and personalized treatment. Despite standard immunochemotherapy curing 60-70% of patients, many experience treatment failure or relapse, and options after second-line therapies remain limited. This observational study examines treatment patterns, demographic, and clinical characteristics of DLBCL patients in the Middle East and Africa. It includes two patient groups those newly diagnosed and eligible for treatment, and those with relapsed or refractory DLBCL who have failed previous therapies. The study aims to explore access to novel therapies and understand real-world treatment approaches in these regions. Participants will be observed over 22 months, during which researchers will document treatment regimens, patient risk profiles, and access to new treatments. Data collection includes patient demographics, disease characteristics, and treatment outcomes. The study does not involve experimental treatments but gathers detailed information to inform future care strategies for DLBCL patients.

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.

This research aims to evaluate the safety and effectiveness of a medical device called ETHIZIA compared to SURGICEL Original for controlling minimal, mild, or moderate soft tissue bleeding during open surgery. The study focuses on bleeding that occurs in areas such as the abdomen, pelvis, thorax excluding the heart, and extremities, where standard methods to stop bleeding are ineffective or impractical. Participants will be randomly assigned to receive either the ETHIZIA patch or SURGICEL Original applied directly to the bleeding site during surgery. Both devices are intended to stop bleeding by achieving hemostasis within 3 minutes and without rebleeding up to 10 minutes after application. After the surgery, participants will be monitored and followed up for 28 days. During the study, participants will undergo assessments to measure the percentage of cases achieving hemostasis at the target bleeding site and the time taken to stop bleeding. Additional evaluations include monitoring for rebleeding, treatment failures, and the need for additional applications or surgical maneuvers. Safety and efficacy data will be collected up to 10 minutes intraoperatively and through follow-up visits within 28 days after surgery.

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 IUdl. 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 11013 vector genomes per kilogram. BBM 002 is designed to increase the bodys 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 SR604, a new drug, to understand its safety, how the body processes it, and its effects in both healthy adults and adults with bleeding disorders such as Hemophilia A, Hemophilia B, or Factor VII deficiency. The study includes healthy volunteers in Part A and patients with these bleeding disorders, some of whom may have inhibitors, in Part B. This first-in-human trial aims to gather important information about SR604s safety and effectiveness. In Part A, healthy participants receive a single subcutaneous injection of SR604 or a placebo in escalating doses across several groups. Part B involves participants with bleeding disorders receiving multiple subcutaneous injections of SR604 every four weeks at increasing dose levels. Both parts involve randomized assignment to treatment or placebo groups, with Part A focusing on single doses and Part B on multiple doses over time. Participants will be monitored for approximately three months, with assessments including medical exams, blood tests, and safety evaluations. Researchers will track adverse events, changes in blood clotting markers, drug concentration levels, bleeding events, and the presence of antibodies against the drug. This monitoring helps understand the drugs effects and safety profile throughout the study period.