Minsk

Researchers are evaluating the long-term safety of study treatments for participants with pulmonary hypertension (PH) who were treated with specific medications in earlier clinical studies and have no other way to access these treatments. This study is designed for people who have finished their previous parent studies and may continue to benefit from the treatment. It is an open-label, phase 3 platform study that follows participants over an extended period to monitor safety. Participants will receive one of the study drugs they used in their parent study, including oral macitentan, selexipag, or a fixed-dose combination of macitentan and tadalafil. Adults take standard doses, while children aged 2 to under 18 years receive doses adjusted for their body weight to match adult exposure levels. Selexipag is given twice daily with dosing adjusted for body weight in children. This treatment continues as in the parent studies, providing ongoing access to these medications. During the study, participants will be regularly monitored for treatment-emergent adverse events, serious adverse events, and any events leading to discontinuation or death, with follow-up lasting up to 84 months. Female participants of childbearing potential will undergo monthly pregnancy testing and agree to contraceptive use during the study and safety follow-up. The study involves ongoing safety assessments to ensure participant well-being while providing access to the study treatments over the long term.

Researchers are evaluating treatment options for children and adolescents with acute lymphoblastic leukemia (ALL) who are in complete remission and need a stem cell transplant with a myeloablative conditioning regimen. This multinational phase III study compares total body irradiation (TBI) combined with chemotherapy to chemotherapy-based conditioning alone. The trial investigates whether chemotherapy conditioning can replace TBI without compromising outcomes, considering the serious late effects of TBI such as organ damage, growth issues, and secondary cancers in growing children. Participants receive different conditioning regimens before their transplant depending on age, donor type, and country-specific practices. Patients aged 4 years and older with matched donors are randomized to receive either fractionated 12 Gy TBI plus etoposide or chemotherapy regimens including fludarabine, thiotepa, and busulfan or treosulfan. Children under 4 years receive chemo-conditioning without irradiation. Those with mismatched donors are assigned based on stem cell source to different conditioning strategies. The study includes a follow-up period to monitor outcomes and late effects. During the study, participants undergo regular assessments including survival monitoring up to 10 years after transplant. Researchers track overall survival and event-free survival depending on donor type and conditioning received. The study also evaluates adverse events and long-term safety. Participants and their families provide consent and agree to follow study procedures at participating centers. This detailed monitoring aims to understand the best conditioning approach balancing efficacy and late complications in pediatric ALL transplant patients.

Researchers are conducting an open-label, prospective, randomized clinical trial to evaluate the effectiveness, tolerability, and safety of a single intraperitoneal dose of the investigational drug Prospidelong in patients with disseminated gastric cancer. The study will include 120 patients aged 18 to 75 years, divided equally into an experimental group receiving Prospidelong and a control group without the drug. This Phase II-III study aims to increase survival over an average of two years. Patients in the experimental group will receive a single intraperitoneal dose of 4000 mg of Prospidelong after surgical placement of a control drainage in the abdominal cavity. Both groups will continue systemic chemotherapy following established clinical protocols for metastatic gastric cancer. The study involves screening, randomization, and treatment periods, with detailed clinical and laboratory assessments performed before and after surgery. Participants will be closely monitored daily during hospitalization and through follow-up visits. Assessments include physical examinations, blood tests, urine analyses, ultrasound or CT scans of the abdomen, ECOG performance scoring, and evaluation of patient complaints. The study will record changes in treatment and adverse events to measure outcomes such as survival and safety throughout the study duration.

Researchers are evaluating the safety and effectiveness of anti-BCMA CAR-T cell immunotherapy in adults with relapsed or refractory multiple myeloma who have had at least two previous treatments. This Phase I/II open-label study aims to assess the safety profile, including possible side effects such as cytokine release syndrome and infections, and the overall response rates like partial and complete responses to the treatment. The treatment involves collecting the patient's own T cells through leukapheresis, followed by a lymphodepleting chemotherapy regimen with fludarabine and cyclophosphamide before receiving a single infusion of anti-BCMA CAR-T cells. Doses range from 50 million to 250 million cells infused on day 0. The study includes a Phase I safety evaluation one month after infusion and a Phase II assessment of treatment response twelve months post-infusion. Participants will be closely monitored throughout the study with assessments including physical exams, laboratory tests, and evaluations for side effects and treatment effectiveness. Researchers will measure safety outcomes in the first month and overall response rates at one year. The study also tracks the duration of response, progression-free survival, and overall survival to better understand the therapy's impact over time.

Researchers are evaluating the efficacy, safety, pharmacokinetics, and immune response to BCD-236 combined with chemotherapy in women with relapsed or metastatic triple negative breast cancer (TNBC). This Phase 2 study focuses on patients who have received at least one prior systemic therapy and whose cancer has progressed or relapsed. The study aims to better understand how this combination treatment works in later lines of therapy for this aggressive breast cancer subtype. Participants will receive BCD-236 as an intravenous infusion along with chemotherapy, which will be chosen at the investigator's discretion. The study compares this combination treatment's effects and monitors participants over time. The primary outcome measured is the overall response rate at 24 weeks after starting treatment, assessing how well tumors respond to the therapy. Throughout the study, participants will undergo tumor assessments using RECIST 1.1 criteria to measure treatment response. Eligibility requires confirmation of AXL expression in tumor cells from fresh or archival tumor samples. Patients will be monitored for safety and disease progression, with evaluations including physical exams and performance status assessments. The study includes women aged 18 to 74 years with adequate health to participate and a life expectancy of at least four months.

Researchers are evaluating the safety and effectiveness of the AccuCinch Ventricular Restoration System in patients with heart failure who have a reduced ejection fraction, a condition where the heart does not pump blood efficiently. This international, randomized, open-label study involves patients diagnosed with heart failure and dilated cardiomyopathy, aiming to compare this device plus standard medical treatment against standard treatment alone. The study focuses on improving heart function and patient quality of life. Participants are randomly assigned to two groups: one receiving the AccuCinch device along with guideline-directed medical therapy, and the other receiving only the medical therapy. About 200 patients will be in each group. Medical treatments are optimized according to current heart failure guidelines. The study includes multiple assessments over time to monitor the device's impact and the patients' heart health. Throughout the study, participants will undergo various tests and evaluations including quality of life questionnaires, six-minute walk tests, and monitoring for major adverse events related to the device or femoral artery access. The study will track outcomes for up to 365 days, measuring deaths, heart failure hospitalizations, and changes in heart function scores. Participants must attend follow-up visits and comply with testing schedules to assess the safety and benefits of the treatment.

Researchers are evaluating the effectiveness and safety of a combination drug containing clotrimazole and lactulose compared to clotrimazole alone in adult women diagnosed with candidal vulvovaginitis. This adaptive clinical trial aims to confirm if the combination treatment provides better clinical and microbiological recovery by Day 25. The study also looks at symptom severity, changes in vaginal Lactobacillus levels, and patient satisfaction with the treatments. Participants were randomly assigned to one of three groups receiving vaginal suppositories or tablets: clotrimazole plus lactulose suppositories, clotrimazole (Canesten) vaginal tablets, or lactulose suppositories alone. Each treatment contains specific doses of the active ingredients, and the study compares these interventions over the course of the trial. During the study, women undergo clinical and microbiological assessments to measure recovery and symptom changes at multiple visits, including Day 25 (Visit 4) as the primary outcome point. Researchers monitor Lactobacillus content and collect patient feedback on treatment satisfaction. Safety and efficacy are observed throughout the trial, which includes adult women aged 18 to 60 years who meet specific health criteria.

Researchers are evaluating the real-life effectiveness, safety, and usage patterns of Octapharma's factor VIII (FVIII) concentrates Nuwiq, Octanate, and Wilate in patients with severe haemophilia A who have either never been treated or have had minimal previous treatment. This study focuses on previously untreated patients (PUPs), often young children, and minimally treated patients (MTPs), to better understand treatment outcomes and inhibitor development in routine clinical practice. Because haemophilia A is rare and treatment practices vary, especially for PUPs, collecting real-world data is important to improve treatment guidelines and benefit-risk assessments. The study observes patients prescribed Octapharma's FVIII concentrates without changing their treatment, capturing how these products are used in everyday healthcare settings. There are no experimental interventions as this is a non-interventional study. The study includes patients starting or continuing treatment with these FVIII concentrates, monitoring their usage, dosing, and frequency as determined by their healthcare providers. Participants are monitored for effectiveness by tracking the annual rate of breakthrough bleeding episodes and safety by recording adverse drug reactions over 100 exposure days. Data on inhibitor development is also collected. Patients of all ages and genders with severe haemophilia A are eligible if they meet treatment history criteria. Informed consent is obtained before data collection. The study gathers information from routine clinical visits and treatment records without additional procedures or interventions.

Researchers are studying Chronic Myeloid Leukemia (CML), a rare blood cancer affecting children and adolescents. This international study aims to better understand the characteristics of CML in young patients under 18 years old, describe current treatment approaches, identify factors that affect prognosis, and develop scoring systems to improve individual treatment decisions. The study also seeks to assess the side effects and long-term impacts of treatments, especially tyrosine kinase inhibitors, on growth and development in this population. This is an observational study collecting both retrospective and prospective data from medical records and existing databases worldwide. It includes patients diagnosed with Philadelphia positive and/or BCR-ABL positive CML at any disease phase and treatment status. Data are managed centrally in France and gathered through national coordinating centers in participating countries, with follow-up data collected twice a year. The study is expected to last about five years starting in August 2009. Participants will provide anonymized information including demographics, medical history, and treatment details. Researchers will monitor and analyze these data to describe patient characteristics, treatment patterns, outcomes, and prognosis factors using statistical methods. Reports will be shared regularly with study contributors and can be presented at scientific meetings. All patient information is handled confidentially under strict ethical guidelines.

Researchers are evaluating the safety and effectiveness of the Myval Transcatheter Heart Valve (THV) Series compared to Contemporary Valves (Sapien THV Series and Evolut THV Series) in patients with severe symptomatic native aortic valve stenosis. This prospective, randomized, multinational, and open-label trial involves 768 participants, evenly divided between the two treatment groups, using a transfemoral approach for valve replacement. Participants will receive either the Myval THV Series or one of the Contemporary Valves, with device sizes ranging from 20 mm to 29 mm for Myval and Sapien series, and 23 mm to 34 mm for the Evolut series. The study includes non-randomized registries for patients requiring extra-large Myval valve sizes and for initial lead-in cases performed under guidance before randomization. Throughout the study, participants will be monitored for safety and effectiveness outcomes, including the Primary Combined Safety and Effectiveness Endpoint defined by the Valve Academic Research Consortium-3 within 30 days post-procedure. Researchers will assess valve function and patient health to compare the performance of the devices, with follow-up evaluations conducted to ensure thorough safety monitoring.