Pontoise

Researchers are evaluating the efficacy, safety, and tolerability of two dosing regimens of itepekimab compared to placebo as an add-on treatment to intranasal corticosteroids in adult men and women with chronic rhinosinusitis with nasal polyps (CRSwNP). This multinational, randomized, double-blind, placebo-controlled Phase 3 study includes participants aged 18 years and older who have inadequately controlled CRSwNP. The study aims to better understand how these treatments impact nasal polyp symptoms and disease control over a one-year period. Participants will be randomly assigned to receive one of two dosing regimens of itepekimab or a placebo, all administered by subcutaneous injection. All participants will continue using mometasone furoate nasal spray as standard intranasal corticosteroid therapy. Treatment will last up to 52 weeks, followed by a 20-week safety follow-up period. The study includes a total of 9 site visits and 20 phone or home visits during the participant's involvement. Participants will be involved in regular assessments including endoscopic nasal polyp scoring and nasal congestion symptom evaluations at baseline and throughout the 24 weeks, among other time points. Researchers will monitor changes in nasal polyp scores and nasal congestion scores to measure the treatment effects. Safety and tolerability will be closely followed during the treatment and safety follow-up periods, with total participation lasting up to 76 weeks for most participants, or 56 weeks for those transitioning to an extension study.

Researchers are evaluating the safety and tolerance of elritercept, a recombinant fusion protein, in adults with anemia linked to lower-risk myelodysplastic syndromes (MDS). The study aims to understand how elritercept affects red blood cell production and to monitor participants for any worsening of MDS during treatment. This is a Phase 2, open-label study focused on patients with very low, low, or intermediate risk MDS. Participants receive elritercept through subcutaneous injections at different dose levels to assess safety and effects. The study includes multiple parts, with initial treatment cycles followed by an extension phase for those who complete the first part without dose-limiting toxicities and may benefit from continued treatment. The study also includes several cohorts based on specific MDS characteristics and transfusion needs. During the study, participants undergo regular evaluations including blood tests, bone marrow assessments, and monitoring for adverse events. Researchers will track the number of treatment-emergent and serious adverse events for up to 11.2 years. Participants are closely monitored for how well they tolerate elritercept and its impact on anemia and red blood cell production throughout the study duration.

Researchers are studying the management and follow-up of non-muscle-invasive bladder cancer (NMIBC), a type of bladder tumor that affects the inner lining and underlying tissue but not the muscle layer. This cancer type accounts for a significant portion of bladder cancer cases in France, with many patients experiencing tumor recurrence within five years. The study aims to evaluate the diagnostic accuracy of urine biomarker tests compared to bladder endoscopy, which is the current standard for detecting tumor recurrence. Additionally, it will describe tumor characteristics, patient history, treatments, and regional differences in care. Patients being monitored for NMIBC and undergoing routine care will have their medical details, including prior treatments and urine test results, recorded in a registry. Follow-up includes regular bladder endoscopy exams, with dates and findings noted by urologists. Urine test results taken before biopsies will also be tracked. This observational study will analyze the performance of urine tests by calculating sensitivity, specificity, and predictive values, and exploring differences based on tumor grade, stage, and previous treatments. The goal is to include 8000 patients across France over six years. Participants will provide data through medical records and routine exams during their personalized care plans. Urine samples and bladder fibroscopy results will be collected at each follow-up visit to assess test accuracy over a five-year period. Researchers will monitor recurrence-free survival and urine test performance, aiming to identify if urine tests can safely reduce the need for invasive cystoscopy. The study focuses on long-term monitoring to better understand and improve care for NMIBC patients.

Premature birth, especially when combined with intrauterine growth retardation, remains a significant public health challenge. This research aims to assess the effects of hospital discharge on newborns weighing less than 2 kilograms, focusing on their short-term and long-term health outcomes. The study includes infants discharged from the neonatology department at the NOVO hospital between 2012 and 2024 and considers advances in neonatal care and parental involvement that have improved survival rates. The study collects data through questionnaires completed by mothers and fathers, either on paper or by phone, regarding their newborns weighing under 2 kilograms from discharge up to 11 months of age. It evaluates the impact of early discharge practices that rely on the infants' physiological readiness and parental support, including home visits by qualified health professionals. The focus is on babies discharged from the neonatology department or the Kangaroo Unit at the NOVO hospital. Participants will be followed up to approximately 11 months after discharge to observe their health status. Data collection includes information from parents and hospital records to track morbidity and mortality outcomes. The study measures the average health outcomes of these premature infants to understand better the consequences of early discharge on this vulnerable population.

This research is focused on patients diagnosed with Atypical Hemolytic-Uremic Syndrome (aHUS). It aims to gather post-marketing safety data for patients treated with the drugs eculizumab or ravulizumab, as well as to monitor the progression of the disease in all participating patients regardless of treatment status. The study is observational and non-interventional, conducted across multiple centers and countries. No specific treatments or interventions are administered as part of this study; rather, it observes patients who have already been treated or untreated with eculizumab or ravulizumab. The study collects data from these patients over time to understand safety outcomes and disease progression. Participants will be followed to record the proportion experiencing certain events over a period of 10 years and the timing of first and subsequent occurrences of these events over 5 years. The study involves gathering clinical information and safety data through routine monitoring. Patients or their guardians provide informed consent and may be asked for assent if applicable. The study tracks long-term outcomes and safety in aHUS patients.

This research aims to characterize the autoimmune T and B lymphocytes involved in the development of type 1 diabetes (T1D) by comparing individuals with T1D, other forms of diabetes or autoimmune conditions, and those without disease. The study hypothesizes that understanding these immune cells will clarify disease mechanisms and help identify new biomarkers for diagnosis, prognosis, and treatment monitoring. Participants in this national multi-center non-interventional case-control cohort study will provide biological samples, including blood and stool specimens. For participants undergoing abdominal surgery with planned lymphadenectomy, lymph node samples will also be collected. This approach allows detailed analysis of immune cell characteristics across different groups. Participants will be followed for up to six years to measure the frequency and phenotype of autoimmune T lymphocytes reactive to islet antigens. Various assessments will include collection and analysis of biological samples and clinical information. Safety monitoring and informed consent will be strictly maintained throughout the study duration.

Researchers are exploring a treatment approach for women with early-stage hormone receptor-positive, HER2-negative breast cancer who face an intermediate risk of cancer recurrence. This phase III trial builds on previous findings that adding the drug ribociclib, a CDK4/6 inhibitor, to standard hormone therapy after surgery can extend the time patients remain free from invasive disease. The study aims to see if using ribociclib allows some patients to avoid chemotherapy and its side effects without compromising treatment effectiveness. Participants will either receive chemotherapy followed by hormone therapy combined with ribociclib or a de-escalated treatment plan that reduces or omits chemotherapy while still using ribociclib and hormone therapy. Ribociclib will be administered for three years as part of the adjuvant treatment after surgery. The study is designed to reflect routine clinical practices for deciding chemotherapy eligibility, using standard pathological assessments and genomic tests. Throughout the trial, women will undergo regular monitoring, including clinical visits, laboratory tests, and heart function assessments, to ensure safety and treatment adherence. Researchers will measure invasive breast cancer-free survival over a period of up to 12 years from randomization. The study also tracks patients' ability to comply with treatment schedules and evaluates long-term outcomes to confirm if chemotherapy can be safely reduced or avoided in this group.

Researchers are evaluating the effectiveness of cotrimoxazole compared to the best standard antibiotic treatment for ventilator-associated pneumonia (VAP) caused by Enterobacteriaceae in adult patients admitted to intensive care units (ICU). This is a multicenter, randomized, non-inferiority Phase 3 trial aiming to show that cotrimoxazole is not worse than standard care in terms of patient survival 28 days after treatment begins. Participants must have a confirmed diagnosis of VAP with bacteria susceptible to cotrimoxazole. Patients are randomly assigned to receive either cotrimoxazole or the best standard antibiotic therapy, which may include beta-lactam or fluoroquinolone antibiotics. Treatment lasts for seven days, starting with an initial appropriate empiric antibiotic therapy. The dosing and administration of antibiotics are tailored according to current ICU guidelines. The trial is open-label due to variable antibiotic regimens in the control group. During the study, patients are closely monitored daily until death, ICU discharge, or 28 days after inclusion. Assessments include vital status, antibiotic use, new infections, and Clostridium difficile infections. Clinical signs and chest X-rays are reviewed on day 7 to evaluate cure. Weekly screenings for multidrug-resistant bacteria are performed until ICU discharge. The vital status is also checked at day 90, with follow-up contact for patients discharged before this time. An independent committee reviews the clinical and radiological outcomes without knowing the treatment assignments.

Researchers are conducting a phase 3, multicenter, open-label, randomized study to evaluate new treatments for adults with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplantation (ASCT). The study aims to compare the effectiveness and safety of a combination of elranatamab and lenalidomide as a replacement for standard chemotherapy during the consolidation phase, and to assess elranatamab alone versus standard care during maintenance therapy. Participants will first receive standard induction therapy with four cycles of a drug combination called D-VRd. After this, they will be randomly assigned to either receive standard consolidation therapy involving high-dose chemotherapy and ASCT followed by D-VRd consolidation (Arm A), or elranatamab combined with lenalidomide for consolidation (Arm B). Upon completing consolidation, patients will be re-randomized to receive maintenance treatment with either lenalidomide alone (Arm C) or elranatamab alone (Arm D). During the study, participants will be monitored for treatment effects including minimal residual disease negativity at the end of consolidation, progression-free survival, and overall survival. The study involves various assessments including clinical evaluations, laboratory tests, and monitoring for disease progression or side effects. The entire treatment and follow-up period may last up to several years, allowing researchers to evaluate long-term outcomes and safety.

Researchers are investigating the interactions between pregnancy and multiple sclerosis (MS), including related disorders such as neuromyelitis optica (NMO) and myelin oligodendrocyte glycoprotein (MOG)-antibody related disorders. This national prospective study aims to better understand how pregnancy impacts MS and associated conditions, focusing on questions about disease-modifying drugs (DMDs), breastfeeding, locoregional analgesia, and the short- and long-term effects on both mother and child. The study is nested within the Observatoire Français de la Sclérose en Plaque (OFSEP) cohort and includes patients across various MS-related diagnoses without age limits. Participants will not receive any experimental treatment but will be observed throughout pregnancy and up to one year after delivery. Their children will be followed until six years of age. The study collects data on pregnancy-related factors, use or discontinuation of DMDs, breastfeeding practices, and analgesia methods during delivery. Neurologists and researchers will gather information on the course of MS and related disorders before, during, and after pregnancy. During the study, participants will be monitored for treatment-related adverse events during pregnancy and the postpartum period, with assessments continuing for up to two years. Data collected will include clinical evaluations and long-term follow-up of both mothers and children. This comprehensive observation aims to provide clearer answers about the safety and impact of therapies and pregnancy-related factors on MS and related neurological conditions over time.