Villefranche Sur Saone

Researchers are creating a national, prospective cohort to study children with idiopathic nephrotic syndrome (INS), a rare kidney disease. The goal is to collect detailed data on patients treated in pediatric nephrology centers across France, Reunion Island, Mayotte, and eventually other French overseas territories. This structured follow-up aims to better understand the disease's characteristics and provide a foundation for future clinical trials. The study involves enrolling pediatric patients diagnosed with INS and systematically collecting clinical, biological, psychological, and social data. Biological samples such as blood, urine, hair, and nails will be gathered at disease onset before immunosuppressive treatment begins. Data will be recorded through medical records from hospital visits and consultations, supplemented by annual telephone interviews for patients without active disease. Quality of life, treatment adherence, and aesthetic impact questionnaires will also be collected and integrated into a secure database. Participants will be followed over at least two years, with data collected regularly by clinical research staff. This includes medical validation of clinical information, annual telephone follow-ups, and questionnaire assessments. The study's primary outcome is the number and characteristics of included cases over two years. This ongoing monitoring will support future nested studies and improve understanding of pediatric INS outcomes and management.

Researchers are conducting a Phase 3 study to compare two front-line treatments for adults with nonsquamous non-small cell lung cancer (NSCLC) that is stage IV or advanced stage IIIB/C. The study focuses on patients whose tumors have a KRAS p.G12C mutation and are negative for PD-L1 expression. The main goal is to evaluate how each treatment affects progression-free survival and overall survival over about 2.5 years. Participants will be randomly assigned to receive either sotorasib combined with platinum doublet chemotherapy or pembrolizumab combined with platinum doublet chemotherapy. Sotorasib is given orally, while pembrolizumab is given intravenously. Both groups will receive the combination therapies as their initial treatment for advanced NSCLC. During the study, participants will be monitored regularly to assess treatment effects and safety. Researchers will track how long patients live without the cancer worsening and overall survival over approximately 2.5 years. The study includes evaluations to determine eligibility and ongoing assessments to monitor health and treatment response throughout the trial period.

Researchers are evaluating the effectiveness and safety of KarXT combined with KarX-EC for treating cognitive impairment in people with mild to moderate Alzheimer's Disease. This Phase 3 study focuses on individuals aged 60 to 85 who have a confirmed diagnosis of Alzheimer's disease according to updated clinical criteria and have specific cognitive scores indicating mild to moderate dementia. Participants will receive either the study drugs KarXT and KarX-EC or a placebo, each given at specified doses on certain days. The study is randomized, double-blind, and placebo-controlled to compare the effects of these treatments on cognitive function. Those already taking certain Alzheimer's medications must have stable doses before and during the study. During the study, participants and their caregivers will attend multiple visits where cognitive assessments and interviews will be performed. Key measures include changes in a cognitive scale (ADAS-Cog11) and clinical impressions of improvement at 24 weeks. Caregivers play an important role by providing information, ensuring medication adherence, and participating in study activities. Safety and treatment effects will be carefully monitored throughout the trial.

Researchers are studying the biological features of advanced ALK-rearranged non-small cell lung cancer (NSCLC) in patients treated with new generation tyrosine kinase inhibitors (TKIs) as their first therapy. This study is part of the national EXPLORE ALK cohort, a multi-center observational project in France, focusing on patients with this specific genetic alteration. The goal is to better understand the tumor biology and resistance mechanisms by analyzing samples from diagnosis through disease progression. The study collects tumor tissue samples at diagnosis and, when possible, at disease progression for RNA sequencing to identify ALK fusion partners, variants, and co-mutations. Blood samples are also taken at diagnosis, first tumor evaluation, and at progression to analyze circulating tumor DNA (ctDNA) using next-generation sequencing panels that detect mutations, fusions, and other genetic changes. These biological analyses are centralized at specialized centers such as the Léon Bérard Center and Rouen University Hospital. Patients are treated with approved ALK inhibitors like alectinib, brigatinib, lorlatinib, or entrectinib as part of their standard care. Participants will provide blood samples at multiple time points and, if possible, tumor biopsy samples for detailed genetic analysis. Researchers will monitor the progression-free survival from treatment start for up to 72 months. The study involves regular evaluations to assess tumor status and collect biological material to track genetic changes over time. Consent for sample collection and participation in the study is required, and patient data is managed within the national health system framework.

Researchers are evaluating the effectiveness of different antimicrobial treatments for infections caused by difficult-to-treat Pseudomonas aeruginosa bacteria. This infection is especially challenging for patients who are critically ill or have weakened immune systems. The study focuses on comparing new beta-lactam/beta-lactamase inhibitor combinations, cefiderocol, and older drugs like aminoglycosides and colistin in real-life clinical settings across multiple hospital centers in France. Participants will receive intravenous antimicrobial therapy tailored to treat their difficult-to-treat P. aeruginosa infection. The study observes the use of new and older antimicrobial drugs to assess their clinical efficacy. Patient data and bacterial samples will be collected and analyzed centrally to better understand drug resistance mechanisms and treatment outcomes. Participants will be monitored for clinical cure shortly after completing therapy and on Day 7 ± 2 days. Researchers will collect clinical information through electronic case-report forms and send bacterial isolates to a national center for detailed testing. Outcomes include cure rates, resistance development, adverse events, and mortality rates, with follow-up during hospitalization and up to 28 days after treatment. The study aims to provide valuable real-world data on treating these challenging infections.

Rare diseases affect about one person in 2,000 and often have a genetic cause, especially when they begin in childhood. This research focuses on rare pediatric autoimmune and autoinflammatory diseases such as systemic lupus, juvenile dermatomyositis, and juvenile idiopathic arthritis. These conditions involve problems with the immune system, either through an inappropriate adaptive immune response or excess innate immune activity. Understanding these diseases is challenging due to their rarity and the limited availability of biological samples. The study involves collecting various biological samples including primary cells (PBMC), DNA, RNA, lymphoblastic lines, and serum. These samples will be used to identify genetic mutations and immunological abnormalities linked to rare autoimmune and autoinflammatory diseases that start in childhood. The intervention includes taking blood samples for genetic analysis and immunological assessments to find biomarkers that can help in diagnosis, prognosis, and monitoring disease activity. Participants, including patients with rare dysimmune diseases and healthy volunteers, will provide blood samples after informed consent. Researchers will analyze these samples to find germline and somatic mutations and specific immunological factors. The study aims to build a biological collection to support various research projects. Outcome measures focus on genetic and immunological findings related to the diseases. Participant involvement includes consent procedures and sample collection, with no specific treatment or follow-up timelines noted.

Researchers are evaluating two antibiotic treatments for adults with bronchiectasis who have an early infection of the airways with Pseudomonas aeruginosa (PA). This infection is linked to more frequent flare-ups, worsened quality of life, and higher risk of death. Current guidelines recommend antibiotic treatment upon the first detection of PA, but the best regimen is unclear. The study aims to compare regimens involving oral fluoroquinolones alone or combined with intravenous and inhaled antibiotics to find effective and manageable treatment options. The study includes two treatment groups over a 3-month period. One group receives a 14-day course of oral ciprofloxacin and inhaled sodium colistimethate followed by 2.5 months of inhaled sodium colistimethate alone. The other group receives a 14-day combination of intravenous ceftazidime, oral ciprofloxacin, and inhaled sodium colistimethate, then continues inhaled sodium colistimethate for 2.5 months. After treatment, both groups are followed for an additional 9 months to monitor outcomes, totaling 12 months of antibiotic therapy and follow-up. Participants will undergo assessments including respiratory sample testing to measure the rate of PA eradication at 6 months. The study will also monitor safety and quality of life during and after treatment. Follow-up visits are scheduled throughout the 12 months to track progress, treatment adherence, and any adverse events. This phase 2 trial aims to provide strong evidence to guide antibiotic use in this patient group, potentially improving medical care and reducing costs.

Researchers are evaluating adults with advanced small cell lung cancer (SCLC) in this study. The main goal is to see if adding a medicine called obrixtamig to the usual treatment, which includes atezolizumab, carboplatin, and etoposide, helps patients live longer compared to the usual treatment alone. Obrixtamig is an antibody-like drug that may support the immune system in fighting cancer. The study also tests a new medical device designed to measure levels of a tumor marker called DLL3. Participants are randomly assigned to one of two groups. One group receives obrixtamig along with the standard treatment, while the other group gets only the standard treatment. All medicines are given through a vein. Those receiving obrixtamig must stay overnight at the study site after their first two treatments with this medicine. Treatment continues as scheduled, following the same general approach for both groups. During up to three years of participation, patients regularly visit the study site for tumor size assessments and health checks. Researchers monitor side effects and compare the results between the two groups to determine treatment effectiveness. The main outcome measured is overall survival over this period.

Researchers are evaluating a paramedical training program for nurses and nursing assistants aimed at improving care for elderly patients aged 75 years and older hospitalized in specialist wards. The study focuses on preventing hospital-related complications (iatrogenesis) and reducing the length of hospital stay, which tends to increase with age. This research is inspired by successful American programs that enhanced geriatric care by training nursing staff, and it aims to adapt these approaches to the French healthcare context. The intervention involves training nurses and nursing assistants using a model similar to hygiene correspondents, supported by Geriatric Mobile Teams (GMT). The GMTs coordinate and support this training to disseminate good geriatric practices systematically. This approach builds on previous programs that emphasized the role of nursing staff in reducing complications like hospital confusion among elderly patients. Participants will be monitored for the length of their hospital stay, calculated from admission to discharge, up to one month. The study will also assess the prevention of hospital-related complications through this training approach. Eligible patients will be tracked during their hospital stay, and researchers will use medical records to measure outcomes. The total participation duration corresponds to the patient’s hospital stay within the study period.

Large-cell neuroendocrine carcinomas (LCNECs) of the lung are rare and aggressive tumors included with small-cell lung cancers in a subgroup of high-grade pulmonary neuroendocrine tumors. Diagnosing LCNEC is challenging, and the prognosis for advanced cases is poor, with survival typically around 8 to 10 months. Researchers are evaluating the effectiveness of combining durvalumab with etoposide and platinum-based chemotherapy as a first-line treatment for advanced LCNEC, based on promising results from related lung and digestive cancer studies. Participants receive treatment with durvalumab combined with etoposide and either carboplatin or cisplatin. The treatment starts with an induction phase consisting of 4 cycles every 3 weeks for 12 weeks, followed by a maintenance phase with durvalumab every 4 weeks for up to 24 months. This open-label, multicenter phase II study uses an external control arm for comparison and aims to assess both efficacy and safety. Throughout the study, participants will undergo regular medical evaluations including imaging scans and laboratory tests to monitor disease progression and treatment effects. The primary outcome measured is the progression-free rate at 12 months. Safety and tolerability will also be monitored. Patients will be followed closely during treatment and maintenance phases, with scheduled visits every few weeks over a period extending up to two years.