Pontoise

Researchers are studying the safety and tolerability of elritercept in adults with anemia linked to very low, low, or intermediate risk Myelodysplastic Syndromes MDS. The study aims to understand how elritercept affects red blood cell production and the progression of MDS, including how well participants tolerate different doses of the drug. This phase 2, open-label trial focuses on anemia associated with lower-risk MDS and evaluates the impact on healthy red blood cell production. Participants receive elritercept as a subcutaneous injection every 4 weeks, with doses ranging from 0.75 mgkg to 5.0 mgkg during an initial period of up to 4 cycles each 28 days. Following this, participants continue treatment with elritercept every 4 weeks for up to 24 cycles, with dose adjustments based on individual response. Different cohorts include participants with or without ring sideroblasts, those requiring red blood cell transfusions, and those with chronic myelomonocytic leukemia CMML. Some participants may enter a long-term extension phase receiving elritercept every 4 weeks for up to about 10 years. During the study, participants undergo regular monitoring including blood tests, assessments of red blood cell parameters, and tracking of adverse events and disease progression. Researchers measure treatment-emergent adverse events, progression to higher-risk MDS or acute leukemia, transfusion independence, and hematologic improvements over up to 11 years. The study includes follow-ups to evaluate the duration and timing of responses, safety, and overall effects on anemia and MDS progression.

Non-muscle-invasive bladder cancer NMIBC is a type of bladder tumor that affects the inner lining of the bladder without invading the muscle layer. This research aims to collect and analyze real-world data on how NMIBC is managed and followed up in clinical practice across France. The study seeks to assess the performance of urine tests used to detect tumor recurrence and to understand variations in treatment and patient outcomes. The study is observational and involves collecting medical data from patients being monitored for NMIBC, including results from urine biomarker tests done before biopsies or resections. Urologists participating in the French Urology Association will record findings from regular bladder endoscopic exams and urine test results during routine care visits. Data analysis will focus on the accuracy of urine tests in predicting tumor recurrence and how this varies by tumor characteristics and prior treatments. Participants will have their medical history, treatments, and urine test results entered into a registry at enrollment. During follow-up visits scheduled as part of their personalized care, data on bladder exams and urine tests will be collected. Researchers will evaluate outcomes such as recurrence-free survival and the diagnostic value of urine biomarkers over a five-year period. The study plans to include around 8,000 patients over six years to improve understanding of NMIBC management and follow-up.

This research aims to evaluate the effects of discharging premature newborns weighing less than 2 kilograms from the hospital. The study focuses on both short-term and long-term outcomes for these infants who were treated at the NOVO hospitals neonatology department between 2012 and 2024. Prematurity and low birth weight are significant public health concerns, and advances in neonatal care along with parental involvement have reduced mortality rates among these vulnerable babies. Participants include premature newborns with intrauterine growth retardation discharged home weighing under 2 kilos. Data is collected through questionnaires completed by mothers and fathers, either on paper or by phone, covering the period from discharge until the baby reaches up to 11 months of age. The study also considers parental support and medical opinions regarding discharge weight criteria. During the study, researchers observe various health indicators including growth in weight, height, and head circumference, as well as morbidity and incidence of Sudden Unexplained Infant Death Syndrome SUIDS. Parents experiences and the support they receive are also assessed remotely. The main outcome is the short-term health status of these newborns at an average of 11 months post-discharge, with additional evaluations at around 6 months. Participation involves completing questionnaires and data collection related to the infants health and development.

This research aims to collect long-term safety and disease progression data on patients diagnosed with atypical hemolytic-uremic syndrome aHUS, including those treated or untreated with the drugs eculizumab or ravulizumab. The study is observational and involves multiple centers and countries, focusing on real-world information after these treatments have been marketed. Participants include patients of any age diagnosed with aHUS, regardless of whether they have identified complement genetic variants or antibodies. The study gathers data without administering new treatments, monitoring patients who may or may not have received eculizumab or ravulizumab. The registry collects information over extended periods to understand safety events and disease course. During the study, researchers track safety-related events over 10 years and the timing of these events within 5 years. Data collection involves reviewing patient health status and disease progression without altering their usual care. The study relies on informed consent and may include minors with appropriate assent. Participation duration varies, with continuous observation to gather comprehensive post-marketing safety data.

Researchers are conducting a national, multi-center observational study to understand the differences in autoimmune T and B lymphocytes among people with type 1 diabetes, other diabetes types or autoimmune conditions, and those without these diseases. The study aims to clarify how these immune cells contribute to type 1 diabetes development and to discover new biomarkers for diagnosis and treatment monitoring. This research is supported by the Institut National de la Sant Et de la Recherche Mdicale in France. Participants are grouped based on their diabetes status or autoimmune conditions, including those with type 1 diabetes, other types of diabetes, no diabetes, or those undergoing lymphadenectomy during abdominal surgery. Biological samples such as blood, stool, and lymph node tissues are collected to analyze immune cell characteristics. This study does not involve any treatment but focuses on detailed immune system profiling over six years. During the study, participants will provide biological samples and undergo evaluations to measure the frequency, phenotype, and activity of autoimmune lymphocytes. Researchers will compare immune cells in blood and pancreatic lymph nodes, identify novel autoimmune targets, and assess links between biomarkers and insulin secretion. The primary outcome is to define autoimmune T lymphocyte features related to islet antigens over six years, with secondary outcomes exploring B lymphocytes and pathogenicity. Participation involves ongoing monitoring and sample collection to better understand type 1 diabetes mechanisms.

Researchers are studying the use of ribociclib, a CDK46 inhibitor, in women with early hormone receptor-positive HR and HER2-negative breast cancer who are at intermediate risk of cancer recurrence. The study aims to see if patients can avoid chemotherapy, which has significant side effects, by using ribociclib along with hormone therapy after surgery. This phase III trial builds on the NATALEE study, which showed that ribociclib added to hormone therapy improved survival free of invasive disease in similar patients. Participants will be randomly assigned to one of two groups one receiving ribociclib plus endocrine hormone therapy, and the other receiving chemotherapy followed by ribociclib and endocrine therapy. Ribociclib treatment lasts for three years, and chemotherapy is given before starting ribociclib and hormone treatment. The trial aims to compare the outcomes of chemotherapy de-escalation versus standard treatment in this patient group. During the study, participants will attend scheduled visits for treatment and monitoring, including laboratory tests, heart monitoring with ECG, and questionnaires about their quality of life. Researchers will track invasive breast cancer-free survival and other outcomes such as overall survival and treatment side effects for up to 12 years. Safety and quality of life assessments will continue during and after treatment to evaluate the long-term effects and benefits of the treatment approaches.

Researchers are investigating the timing of diagnosis and management of Gestational Diabetes Mellitus GDM in pregnant women, focusing on whether early intensive treatment starting before 20 weeks of pregnancy is better than the usual approach of waiting until 24 to 28 weeks. The study addresses concerns that early fasting plasma glucose testing may not accurately predict later GDM and examines if early intervention improves outcomes for mothers and babies. This is a randomized trial comparing two management strategies for early GDM, a condition characterized by high blood sugar during pregnancy. The trial compares two groups of pregnant women diagnosed with early GDM. One group receives intensive metabolic treatment immediately after diagnosis, including diet, physical activity, blood glucose monitoring, and possibly insulin therapy following French guidelines. The other group follows the standard approach with no intervention until GDM screening at 24 to 28 weeks, after which treatment begins if needed. The study continues treatment until delivery. Participants will be monitored throughout pregnancy with various metabolic tests, including fasting plasma glucose, HbA1c, and oral glucose tolerance tests. Researchers will track the frequency of maternal and fetal complications at delivery and assess insulin use, metabolic parameters, and neonatal outcomes up to six weeks postpartum. Additional analyses include studying microbiota and breast milk composition. The total participation period spans from early pregnancy until six weeks after delivery, with detailed data collected at multiple time points.

Researchers are evaluating the effectiveness of cotrimoxazole compared to standard antibiotic therapy for treating ventilator-associated pneumonia VAP caused by Enterobacteriaceae in adult patients in intensive care units ICU. This multicenter randomized trial aims to determine if cotrimoxazole is not inferior to the best standard care in terms of survival 28 days after treatment begins. Patients with confirmed VAP and susceptibility to cotrimoxazole are included, with specific attention to those with or without septic shock and COVID-19 pneumonia. Participants are randomly assigned to receive either cotrimoxazole or standard antibiotic treatment, such as beta-lactams or fluoroquinolones, for a total of 7 days including initial empiric treatment. The antibiotic dosing and administration are adjusted based on current ICU recommendations. The treatment period can last up to 28 days or until death or ICU discharge if earlier. The study is not blinded due to variable therapies in the control group. During the study, patients are monitored daily until death, ICU discharge, or day 28 for vital signs, antibiotic use, new infections, and Clostridium difficile infection. Clinical and radiological evaluations occur at day 7, and weekly screening for multidrug-resistant bacteria is performed. Survival status is followed up to 90 days through phone or hospital interview. An independent committee reviews clinical and radiological outcomes without knowledge of treatment assignment to ensure unbiased assessment.

Researchers are conducting a phase 3, open-label, randomized study in adults aged 18 to 69 with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplantation ASCT and high-dose chemotherapy. The trial aims to evaluate the effectiveness and safety of using elranatamab and lenalidomide as alternatives to standard chemotherapy in the consolidation phase, and to compare elranatamab alone with standard maintenance therapies. This study involves about 824 patients across approximately 70 sites and addresses important outcomes like minimal residual disease MRD negativity, progression-free survival, and overall survival. Participants receive standard induction therapy with four cycles of D-VRd daratumumab, bortezomib, lenalidomide, dexamethasone followed by either ASCT and D-VRd consolidation standard arm or elranatamab with lenalidomide consolidation experimental arm. After consolidation, patients enter a maintenance phase where they are re-randomized to receive either daratumumab plus lenalidomide or elranatamab monotherapy for about two years. Treatment adjustments depend on MRD status and disease progression during maintenance. Throughout the study, participants undergo regular assessments including disease evaluations and safety monitoring. Researchers measure MRD negativity rates, progression-free survival, overall survival, adverse events, and quality of life using validated questionnaires. The trial offers long-term follow-up, lasting up to around 10 years, to capture comprehensive data on treatment effects and patient well-being.

This research aims to better understand how pregnancy affects women with multiple sclerosis MS and related neurological conditions, including neuromyelitis optica and myelin oligodendrocyte glycoprotein antibody-related disorders. The study focuses on important questions about the use of disease-modifying drugs during pregnancy, the effects on the mothers health and disability, the childs health, breastfeeding, and pain management during delivery. It is a national, prospective, observational study conducted within the French MS cohort called Observatoire Franais de la Sclrose en Plaque OFSEP. Women with MS or related disorders who are pregnant will be followed throughout their pregnancy and for one year after giving birth. Their children will also be monitored until they reach six years of age. There is no intervention or treatment given as part of this study instead, it collects detailed information on the course of MS during pregnancy and postpartum, including any use or discontinuation of disease-modifying drugs and other pregnancy-related factors. Participants will have regular follow-ups during pregnancy and postpartum to assess their health and any treatment-related side effects. Researchers will collect data on relapses, disability progression, breastfeeding, and use of pain relief during delivery. Children born to these women will be observed for up to six years to evaluate long-term outcomes. The main outcomes measured include the number of treatment-related adverse events during pregnancy and postpartum, helping to improve knowledge for neurologists and families regarding MS and pregnancy.