Minsk

This research aims to provide continued access to study treatments for participants with pulmonary hypertension who have completed previous clinical studies but have no other way to receive these treatments. It focuses on assessing the long-term safety of the study drugs macitentan, selexipag, and a fixed dose combination of macitentan and tadalafil. The study is designed for participants who benefited from these treatments during parent studies and have no alternative approved therapies available. Participants will continue receiving oral study drugs macitentan once daily with doses adjusted for children by weight, selexipag twice daily with dosing based on previous maintenance dose and body weight for children, or a fixed dose combination of macitentan and tadalafil once daily. Adults will have study visits every six months, while pediatric participants will have visits every three months. These visits will collect information on the treatments safety and effectiveness until the participant stops treatment, the drug becomes commercially available, or the study ends prematurely. During the study, participants will undergo regular assessments to monitor safety, including tracking treatment-emergent adverse events, serious adverse events, events leading to discontinuation, and deaths for up to 84 months. Visits will include collecting efficacy and safety data to ensure ongoing monitoring. The overall participation duration may extend for several years, allowing careful observation of long-term effects and safety of these treatments in people with pulmonary hypertension.

Researchers are evaluating the tolerability, safety, and effectiveness of a gene therapy called GNR-097 for boys aged 4 to 9 years with Duchenne muscular dystrophy DMD. The study includes two phases Phase I involves two dose groups receiving increasing amounts of the gene therapy, and Phase II is a randomized, single-blind, placebo-controlled study where some participants receive placebo initially with the option to receive gene therapy in the second year. Participants will receive a single intravenous infusion of GNR-097 on Day 1 during Phase I. In Phase II, participants randomized to placebo will receive a matching placebo infusion on Day 1, then have the option to receive the gene therapy infusion at the start of the second year. The study aims to determine the best dose in Phase I and assess safety and efficacy in Phase II with placebo control. Throughout the study, participants will be monitored for adverse events and safety from baseline through Week 104. Researchers will evaluate treatment-emergent adverse events, serious adverse events, and adverse events of special interest. Participants will undergo assessments of functional activity and immune response, and their health will be tracked during screening, treatment, and follow-up periods. The total study duration spans up to approximately four years from the start date in 2025 to completion in 2029.

Researchers are studying children and adolescents with acute lymphoblastic leukemia ALL who are in complete remission and require allogeneic hematopoietic stem cell transplantation HSCT. This multinational phase III trial aims to optimize therapy by comparing chemotherapy-based conditioning regimens to total body irradiation TBI combined with chemotherapy. The study focuses on evaluating overall survival and event-free survival, considering the risks of late side effects like organ dysfunction, growth issues, hormonal problems, and secondary cancers from TBI. Participants receive one of several conditioning regimens before HSCT. These include TBI with etoposide VP16 for patients older than 4 years, or chemotherapy combinations such as fludarabine, thiotepa, treosulfan, intravenous busulfan, or busulfan with VP16 and cyclophosphamide, depending on age and donor compatibility. Some patients receive additional medications like ATG Thymoglobulin or Grafalon. The study also stratifies patients by donor type, including matched sibling, matched unrelated, and mismatched donors, to assess outcomes across these groups. During the study, participants undergo regular follow-up for up to 10 years to monitor overall survival, event-free survival, relapse, toxicity, and transplant-related mortality. Data are collected annually starting 18 months after the first patient enrollment. The trial includes assessments of acute and late side effects, with ongoing observation to understand the long-term impact of different conditioning regimens in pediatric ALL patients undergoing HSCT.

Researchers are evaluating the efficacy, tolerability, and safety of a single intraperitoneal dose of the investigational drug Prospidelong in patients with disseminated gastric cancer. This open-label, prospective, randomized clinical study plans to include 120 patients aged 18 to 75 years, divided evenly into two groups one receiving Prospidelong and the other serving as a comparison group. The study aims to assess survival and other health outcomes over an average of two years. Participants receive one dose of Prospidelong 4000 mg, equivalent to 2000 mg of prospidium chloride administered directly into the abdominal cavity after diagnostic surgery. The control group undergoes diagnostic surgery followed by systemic chemotherapy according to approved clinical protocols. After treatment, all patients continue with standard care and chemotherapy as specified by national guidelines. During the study, patients are monitored daily throughout their hospital stay and during follow-up visits. Assessments include physical exams, blood tests, urine analyses, ultrasound or CT scans, and evaluation of performance status using the ECOG scale at multiple time points up to two years post-surgery. Researchers track safety, treatment tolerability, and survival as primary outcomes, with imaging and laboratory tests as secondary measures.

Researchers are studying anti-BCMA CAR-T cell immunotherapy in adults with relapsed or refractory multiple myeloma. This Phase III open-label trial aims to evaluate the safety, tolerability, and effectiveness of this treatment in patients who have received at least two prior therapies and have resistant disease. The study will explore important safety concerns such as cytokine release syndrome, neurological side effects, infections, and blood cell changes, as well as treatment response and survival outcomes. The treatment process involves collecting the patients own T cells through leukapheresis, followed by chemotherapy preconditioning with fludarabine and cyclophosphamide. After this conditioning, a single infusion of the anti-BCMA CAR-T cells is given. Dosages range from 50 million to 250 million CAR-T cells. The study is divided into Phase I focusing on safety and pharmacokinetics and Phase II concentrating on overall response rates and long-term outcomes. Participants will be monitored closely with safety assessments for one month after infusion and followed up for treatment response and survival for up to three years. Researchers will measure how well the CAR-T cells expand and persist in the body, track progression-free and overall survival rates, and observe the duration of response. Trial visits will include clinical evaluations and laboratory tests to assess organ function and disease status throughout the study period.

Researchers are evaluating the effectiveness, safety, how the body processes, and immune response to BCD-236 combined with chemotherapy in women with relapsed or metastatic triple negative breast cancer TNBC who have received previous treatments. This is a Phase 2 study that compares BCD-236 plus chemotherapy versus chemotherapy alone in patients whose cancer has returned or progressed after earlier therapies. Participants will be randomly assigned to one of two groups one receiving BCD-236 by intravenous infusion plus chemotherapy chosen by the investigator, and the other receiving chemotherapy alone. Treatment continues until the cancer worsens, side effects become intolerable, or the study ends. During the study, participants will undergo regular assessments including tumor measurements and health evaluations to monitor response and side effects. The main outcome measured is the overall response rate at 24 weeks, with secondary outcomes including progression-free survival, overall survival, disease control rate, time to response, and duration of response assessed at various times up to 102 weeks. Participants will be monitored closely throughout their treatment and follow-up periods.

Researchers are evaluating the safety and effectiveness of a combination drug containing clotrimazole and lactulose compared to clotrimazole alone in adult women diagnosed with candidal vulvovaginitis. The study aims to determine if the combination drug provides better clinical and microbiological recovery by Day 25 of treatment. This trial uses an adaptive design to assess how lactulose affects fungal infections in the vagina. Participants are randomly assigned to one of three groups one receiving vaginal suppositories with clotrimazole 100 mg and lactulose 300 mg, another receiving vaginal tablets with clotrimazole 100 mg alone, and a third group receiving vaginal suppositories with lactulose 300 mg alone. The study evaluates outcomes at several visits Day 8, Day 15, and Day 25, focusing on symptom improvement, microbiological response, and changes in vaginal bacteria. During the study, participants will undergo clinical assessments and laboratory tests to measure symptom severity and vaginal bacterial content, including molecular analysis of Lactobacillus levels. Patient satisfaction with the therapy will also be recorded. Safety and treatment responses will be monitored throughout the 25-day period, with the main result being the proportion of patients who achieve full clinical and microbiological recovery by Day 25.

This research aims to evaluate the real-life effectiveness, safety, and usage patterns of Octapharmas factor VIII FVIII concentratesNuwiq, Octanate, and Wilatein patients with severe haemophilia A who have either never been treated or have had minimal treatment. The study focuses on previously untreated patients, often young children, and minimally treated patients, gathering more data on treatment outcomes and inhibitor development to better understand optimal treatment approaches in routine clinical practice. Participants receive one of the three FVIII concentrates Nuwiq recombinant FVIII, Octanate plasma-derived FVIII, or Wilate plasma-derived FVIII combined with von Willebrand factor. The study observes how these products are used, including dosing and frequency, without altering prescribed treatments. This non-interventional study collects data during routine care to assess product utilization, safety, and effectiveness, including responses to surgical prophylaxis. During the study, researchers monitor participants for breakthrough bleeding rates and any adverse drug reactions over 100 exposure days to assess treatment effectiveness and safety. They also collect information on FVIII dosing and physicians evaluations of surgical prophylaxis effectiveness. The study involves patients of all ages and follows them through their usual clinical visits, with data gathered via observation rather than treatment changes. The study is designed to provide valuable real-world evidence on these FVIII concentrates in severe haemophilia A patients.

Researchers are studying chronic myeloid leukemia CML, a rare blood cancer, in children and adolescents under 18 years old. This international observational study aims to better understand the characteristics, treatment approaches, and prognostic factors of pediatric CML. It also seeks to evaluate the long-term effects of treatments, especially tyrosine kinase inhibitors, on growth and development in this young population. The study collects both retrospective and prospective data from medical records and databases, without administering new treatments or interventions. Data gathered include demographic details, diagnosis information, treatment histories, and follow-up outcomes. National coordinating centers in participating countries gather anonymized patient data and send it to a central data center in France for analysis. Participants data are collected and updated twice yearly, with ongoing monitoring to describe patient characteristics and treatment policies. Researchers analyze survival, response to treatment, side effects, and long-term outcomes. The study is expected to continue for about five years, with results shared regularly with participating centers and published anonymously.

Researchers are comparing the safety and effectiveness of the Myval Transcatheter Heart Valve THV Series with other contemporary valve devices, specifically the Sapien THV Series and Evolut THV Series, in patients with severe symptomatic native aortic valve stenosis. This prospective, randomized, multinational trial aims to determine if the Myval THV Series performs at least as well as these established valves for treating this condition. The study will enroll a total of 768 participants, with equal numbers receiving either the Myval THV or the contemporary valves. Participants will receive heart valve replacements via the transfemoral approach using valves sized appropriately for their anatomy. The Myval THV Series includes several device sizes ranging from 20 mm to 29 mm, while the comparator devices include Sapien and Evolut valves in various sizes. The trial also features a non-randomized nested registry to evaluate extra-large Myval valves 30.5 mm and 32 mm and a lead-in phase where investigators perform initial implantations to ensure protocol adherence before enrolling study subjects. Throughout the study, participants will be closely monitored with assessments of valve safety and effectiveness using criteria defined by the Valve Academic Research Consortium-3 VARC-3. Outcomes measured include mortality, stroke, kidney injury, bleeding, valve function, device success, and quality of life, among others, followed up to 10 years. The study includes detailed safety and efficacy evaluations at multiple time points, including pre-discharge, 30 days, 1 year, and longer-term follow-ups. Participants will undergo clinical evaluations, echocardiography, and quality of life questionnaires during these visits.