Villefranche Sur Saone

Researchers are conducting a prospective, multicenter cohort study to follow children with idiopathic nephrotic syndrome INS, a rare kidney disease. The study aims to collect data on pediatric patients treated by pediatric nephrologists in France and its overseas territories to better understand the diseases characteristics and support future clinical trials. The study involves regularly recording medical, biological, psychological, and social data through routine clinical follow-ups, hospitalizations, and consultations. Additionally, annual telephone interviews will be conducted for patients in remission. Quality of life, treatment adherence, and treatment impact questionnaires will also be collected. A biobank is established to collect blood, urine, hair, and nail samples at the disease onset before immunosuppressive treatment begins. Participants will be followed from disease onset until age 18 or transfer to adult nephrology care. Data is collected via a secure website, medically validated and entered by clinical research staff. The main outcome is the number of cases included and their characteristics over two years. Participation involves routine care visits, interviews, and questionnaires, with continued monitoring planned through the study period ending in 2048.

Researchers are studying two combination treatments as front-line therapies for adults with stage IV or advanced stage IIIBC nonsquamous non-small cell lung cancer NSCLC that carries a KRAS p.G12C mutation and is negative for PD-L1. This phase 3 trial compares progression-free survival and overall survival in participants receiving either sotorasib with platinum doublet chemotherapy or pembrolizumab with platinum doublet chemotherapy. The goal is to evaluate which treatment combination may better manage this specific lung cancer type. Participants receive one of two treatment combinations sotorasib taken orally with carboplatin and pemetrexed, or pembrolizumab given intravenously with carboplatin and pemetrexed. These therapies are administered as front-line treatments. The study is randomized and open-label, meaning participants are assigned to a treatment group by chance and both participants and researchers know which treatment is given. During the study, participants will be monitored regularly for disease progression and survival over approximately 2.5 years. Additional assessments include quality-of-life questionnaires focusing on symptoms like dyspnea, cough, and chest pain, as well as physical function and global health status up to week 12. Safety is evaluated by tracking adverse events, vital signs, and laboratory test changes throughout the study, which may last up to about 5.5 years including follow-up. Participants receive care under medical supervision while contributing to important research on lung cancer treatment.

Researchers are investigating the efficacy and safety of KarXT combined with KarX-EC for treating cognitive impairment in people with mild to moderate Alzheimers Disease. This phase 3, randomized, double-blind, placebo-controlled study aims to assess how well this combination works compared to placebo in improving cognitive function and overall condition in affected adults aged 60 to 85 years. Participants will receive either the active drugs KarXT plus KarX-EC or a placebo following a specified dosing schedule. The study lasts 24 weeks, during which participants will be randomly assigned to one of these two groups. The trial is designed to carefully monitor effects and side effects of the treatments under controlled conditions. Throughout the study, participants and their caregivers will attend regular visits where cognitive tests like the ADAS-Cog11 and CIBIC will be conducted to track changes from baseline. Additional assessments include daily living activities, neuropsychiatric symptoms, safety labs, vital signs, ECGs, and adverse event monitoring. Caregivers will help report on participant condition and medication adherence, supporting comprehensive evaluation of the treatments impact over the 24-week period.

Researchers are studying advanced non-small cell lung cancer NSCLC with ALK gene rearrangement treated with next-generation tyrosine kinase inhibitors TKIs as first-line therapy. This prospective study, part of the national EXPLORE ALK cohort, aims to understand the biological characteristics and resistance mechanisms of this cancer type. It involves patients with stage IIIB or IV NSCLC who are not eligible for curative locoregional treatment and have confirmed ALK rearrangement. The study collects tumor tissue samples at diagnosis and at disease progression, if available, to analyze ALK fusion partners, variants, and co-mutations using RNA sequencing. Blood samples are taken at diagnosis, first tumor evaluation, and disease progression to analyze circulating tumor DNA ctDNA with next-generation sequencing. Treatments studied include alectinib, brigatinib, lorlatinib, and entrectinib, either marketed or under early access programs. Participants provide blood samples and allow use of tumor tissue for centralized biological analyses. Researchers measure progression-free survival up to 72 months as the primary outcome, along with overall survival, response rates, duration of response, ctDNA clearance, and resistance mechanisms associated with treatment and ALK fusion partners. The study involves regular evaluations over several years to monitor treatment outcomes and biological changes.

Researchers are evaluating different antimicrobial therapies for infections caused by difficult-to-treat Pseudomonas aeruginosa, a type of bacteria resistant to many common antibiotics. This study focuses on comparing new beta-lactambeta-lactamase inhibitor combinations, cefiderocol, and older treatments like aminoglycosides and colistin to understand their effectiveness in real-life settings for patients facing these challenging infections. The study is a prospective, multicenter cohort involving patients who require intravenous antibiotic treatment for their P. aeruginosa infections. Participating patients will receive one of the available antimicrobial options, and their bacterial samples will be analyzed centrally to assess susceptibility and resistance mechanisms. The study collects clinical data throughout treatment and monitors outcomes such as cure rates, resistance development, adverse events, and mortality. Participants will be followed until hospital discharge and up to 28 days after treatment completion. Researchers will track clinical cures, microbiological eradication, adverse events, emergence of resistance, and acquisition of other multidrug-resistant bacteria. Data collection includes electronic case reports and laboratory testing on bacterial isolates. The total participation duration varies according to hospital stay and follow-up schedules, with rigorous monitoring of safety and treatment outcomes.

Rare diseases affect about one in 2,000 people, including approximately three million in France, and many are genetic, often beginning severely in childhood. This research focuses on rare pediatric autoimmune and autoinflammatory diseases such as systemic lupus, juvenile dermatomyositis, and juvenile idiopathic arthritis. These diseases involve either the bodys adaptive immune system attacking itself or an excessive innate immune response, and the exact causes remain not fully understood. Recent advances have improved diagnosis, especially in early, familial, and syndromic cases, but studying these conditions is challenging due to their rarity and limited biological samples. The study aims to build a biological collection of samples including primary cells, DNA, RNA, lymphoblastic lines, and serum. This collection will support various research projects to identify genetic and immunological abnormalities linked to these rare diseases. Participants include minors or adults with rare dysimmune diseases starting in childhood or with familial or syndromic forms, as well as healthy volunteers, with no upper age limit but weighing over 5 kg. Participants provide blood samples for genetic analysis and immunological assessments to identify mutations and biomarkers related to disease diagnosis, prognosis, and activity. Researchers will measure mutations responsible for these diseases and assess disease activity using tools like the Systemic Lupus Erythematosus Disease Activity Index and biomarkers including anti-double stranded DNA and interferon levels. The study includes consented patients and healthy volunteers affiliated with social security, with ongoing monitoring for safety and comprehensive data collection throughout the study period.

Researchers are studying adults with bronchiectasis who have a recent infection of Pseudomonas aeruginosa PA in their airways. This infection is linked to more frequent worsening of symptoms, lower quality of life, and a higher risk of death. The study compares two antibiotic treatment plans to find out if a simpler oral and inhaled antibiotic combination works as well as a more intensive treatment involving intravenous antibiotics. This research aims to provide clearer evidence to guide how these infections should be treated for better health and economic outcomes. The study tests two treatment approaches over a 3-month period. One group receives an oral fluoroquinolone antibiotic combined with inhaled colistimethate sodium, starting with 14 days of both followed by 2.5 months of inhaled antibiotic alone. The other group gets intravenous beta-lactam antibiotic plus the same oral and inhaled antibiotics in the initial 14 days, then continues inhaled treatment alone for 2.5 months. After treatment, participants are followed for 9 more months to monitor their health and infection status, totaling 12 months from the start of therapy. Participants will have regular check-ups during and after treatment to measure how well PA is eradicated and to track any return of infection or worsening symptoms. Questionnaires will assess quality of life and treatment burden at the start, 3 months, and 12 months. Researchers will also monitor antibiotic resistance, adverse events, and healthcare costs over one year. This comprehensive follow-up helps evaluate both the medical and economic impact of the two antibiotic regimens in managing early PA airway infection in bronchiectasis.

Researchers are evaluating treatments for adults with advanced small cell lung cancer SCLC to see if adding a study medicine called obrixtamig to standard therapy improves survival compared to standard treatment alone. Obrixtamig is an antibody-like molecule designed to help the immune system fight cancer. The study also tests a new medical device to measure a tumour marker called DLL3 as part of the evaluation. Participants are randomly assigned to one of two groups one receives obrixtamig plus the standard treatment of atezolizumab, carboplatin, and etoposide, while the other group receives only the standard treatment. All treatments are given through infusions into a vein. Those receiving obrixtamig stay overnight at the study site after the first two doses. This phase III trial compares outcomes between these groups to assess treatment effects. Participants remain in the study for up to three years, with regular visits to the study site. Doctors monitor tumour size and overall health, watch for side effects, and record any unwanted reactions. The main measurement is overall survival over 36 months. Other assessments include progression-free survival, symptom changes, response rates, and safety events. This extensive monitoring helps researchers understand the treatments impact and safety.

Researchers are studying the impact of a special training program for nurses and nursing assistants on elderly patients aged 75 and over who are hospitalized in specialized wards. The goal is to see if this training, adapted from a successful American nursing program called NICHE, can improve the care of elderly patients by reducing hospital-related complications and shortening the length of hospital stays. This training involves teaching healthcare staff about better geriatric care practices, especially to prevent confusion and other hospital-related issues common in older patients. The study compares two groups of patients one group admitted after the introduction of specially trained geriatric nurses and nursing assistants, and a control group admitted before this training was introduced. The training program is coordinated by Geriatric Mobile Teams GMT, who support and guide the nursing staff, helping to spread good geriatric care practices similar to hygiene correspondents in hospitals. The program is designed to improve overall hospital care for older patients by focusing on evidence-based practices. Participants length of hospital stay is measured as the number of days from admission to discharge, with data collected up to one month after admission. The study observes patients during their hospital stay to evaluate the effects of the training program on preventing complications related to hospitalization. The study spans from May 2023 to July 2027. Researchers assess patient outcomes based on medical records, including complications and duration of hospitalization, to determine if this nursing training improves care for elderly hospital patients.

Large-cell neuroendocrine carcinomas LCNECs of the lung are rare, aggressive tumors with poor prognosis and limited treatment options. Researchers are evaluating the combination of durvalumab with etoposide and platinum chemotherapy as a first-line treatment to improve the progression-free rate at 12 months in patients with advanced LCNEC confirmed by expert pathologist review. This phase II, multicenter, open-label study aims to assess the efficacy and safety of this combination compared to historical data. Participants will receive durvalumab combined with etoposide and either cisplatin or carboplatin. Treatment includes an induction phase with cycles every three weeks for 12 weeks four cycles, followed by a maintenance phase with durvalumab every four weeks for up to 24 months. This prospective study uses an external control arm for comparison and focuses on first-line treatment of advanced LCNEC. During the study, patients will be closely monitored through scheduled visits and assessments, including radiological evaluations to measure disease progression. Researchers will track the progression-free rate at 12 months as the primary outcome and also evaluate objective response rates, disease control rates, overall survival, and safety profiles over a period of up to 63 months. Participants will be followed for adverse events and treatment-emergent effects for up to 27 months and 90 days post-treatment, ensuring comprehensive safety and efficacy evaluation.