Toulon

Researchers are investigating metastatic colorectal cancer mCRC patients who have a specific genetic change called the BRAFV600E mutation. This rare subtype of mCRC often shows poor response to current treatments and has a generally poor outlook. The study aims to collect detailed clinical data and biological samples to better understand this condition, including how patients respond to treatments and what factors predict their survival. It focuses on real-world treatment outcomes and biological markers that might influence therapy choices and resistance. Participants will provide blood samples at multiple times during their treatment, including before and during the first three treatment cycles, at 3 and 6 months after starting each treatment line, and when disease progression occurs following certain therapies. The study gathers up to 390 mL of blood per participant over time to analyze circulating tumor DNA and immune environment factors. This observational approach will help researchers identify biomarkers related to treatment response and disease progression. During the study, participants clinical progress and survival will be tracked for up to five years. Researchers will review overall survival from diagnosis to death and assess how prognostic markers relate to progression-free survival and response to treatments. The study involves collecting tumor tissue samples and blood tests, along with routine follow-up visits. All data collected will contribute to understanding BRAFV600E mCRC and improving future treatment strategies.

Researchers are evaluating the efficacy and safety of rilvegostomig compared to pembrolizumab, both combined with platinum-based doublet chemotherapy, as a first-line treatment for patients with locally advanced or metastatic non-squamous non-small cell lung cancer NSCLC whose tumors express PD-L1 at levels of 1% or higher. This Phase III, randomized, double-blind, global study aims to compare these treatments to improve outcomes for this patient group. Participants will receive either rilvegostomig or pembrolizumab, each given intravenously on Day 1 of every 21-day cycle, combined with platinum-based doublet chemotherapy either carboplatin or cisplatin also given on Day 1 of each cycle for up to four cycles. After chemotherapy cycles, patients continue with rilvegostomig or pembrolizumab monotherapy combined with pemetrexed maintenance. The study follows patients for up to approximately six years to monitor treatment effects and safety. During the study, participants undergo assessments including imaging scans to measure tumor size, blood tests to evaluate organ function, and questionnaires about symptoms and quality of life. Researchers monitor overall survival and progression-free survival as primary outcomes, alongside other measures such as response duration and physical functioning. Safety is closely observed throughout, with study visits scheduled regularly during treatment and follow-up periods, lasting up to six years in total.

Researchers are evaluating the safety and effectiveness of rilvegostomig combined with platinum-based chemotherapy compared to pembrolizumab combined with platinum-based chemotherapy as a first treatment for patients with locally advanced or metastatic squamous non-small cell lung cancer mNSCLC whose tumors express programmed death-ligand 1 PD-L1. This Phase III global study focuses on patients with PD-L1 tumor cell expression of 1% or higher and aims to determine which treatment provides better overall and progression-free survival. Participants will be randomly assigned to one of two study groups one group will receive rilvegostomig plus carboplatin and either paclitaxel or nab-paclitaxel chemotherapy, while the other group will receive pembrolizumab plus the same chemotherapy options. Rilvegostomig and pembrolizumab are both given intravenously on Day 1 of each 21-day cycle, with chemotherapy given up to 4 cycles. Nab-paclitaxel may be administered on Days 1, 8, and 15 of each cycle. Treatment continues with rilvegostomig or pembrolizumab until disease progression or other criteria are met. During the study, participants will undergo regular assessments including imaging scans to measure tumor response, laboratory tests to monitor organ function, and patient questionnaires about physical function and quality of life. Researchers will track overall survival, progression-free survival, response rates, and duration of response for up to approximately 6 years. Safety and immune response to rilvegostomig will also be evaluated. Participants will be closely monitored throughout the treatment and follow-up periods.

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 patients with completely removed non-small cell lung cancer NSCLC who have common mutations in the EGFR gene, specifically Del19 or L858R. The study aims to use broad-panel centralized next-generation sequencing NGS testing at the start to better understand molecular factors that predict outcomes and to investigate mechanisms of resistance to the drug osimertinib after treatment. This research focuses on patients with stage pIB to IIIA NSCLC who have undergone thorough preoperative assessments and quality surgical excision reviews. Participants will have plasma circulating tumor DNA ctDNA tested at various stages before surgery optional, 4 to 8 weeks after surgery, before starting adjuvant chemotherapy or osimertinib if given, every 6 months during follow-up, and at relapse if it occurs. Formalin-fixed paraffin-embedded FFPE tissue blocks from surgery and relapse optional will also be collected for molecular analyses. Treatment with osimertinib as adjuvant therapy is planned for up to 3 years, with chemotherapy given beforehand if needed. During the study, patients will be regularly followed every 3 to 6 months with standard care visits and molecular testing. Researchers will monitor the feasibility of the testing approach over 18 months and track the incidence and severity of any side effects related to osimertinib for about 3 years. This ongoing monitoring aims to better understand relapse mechanisms and improve treatment options in metastatic relapse. The total participation duration aligns with the follow-up and safety monitoring periods outlined.

Researchers are studying real-world patient characteristics, treatment patterns, and both short- and long-term outcomes in people with symptomatic obstructive hypertrophic cardiomyopathy HCM across the United States and Europe. The study focuses on patients receiving mavacamten, other treatments for obstructive HCM, or no treatment due to intolerance or prior treatment failure. The U.S. portion evaluates the safety of mavacamten in this setting, while the European part assesses both its effectiveness and safety. Participants receive treatments as part of standard care, either mavacamten or other medications such as beta-blockers, non-dihydropyridine calcium channel blockers, or disopyramide. Treatments are prescribed by physicians according to routine clinical management. The study observes outcomes over time without altering prescribed care. During the study, participants are monitored for changes in heart function, symptoms, and adverse events through clinical assessments including echocardiography and patient-reported questionnaires. Researchers evaluate heart failure events, heart function measures like left ventricular outflow tract gradient and ejection fraction, arrhythmias, hospitalizations, mortality, and quality of life scores. Data is collected at baseline and followed for up to five years to understand real-world treatment effects and safety.

Researchers are studying the impact and burden of three skin conditions moderate or severe alopecia areata, non-segmental vitiligo, and moderate to severe hidradenitis suppurativa. The study includes adolescents and adults and aims to understand how these conditions affect quality of life and daily functioning in a large global population. This is an observational study where participants with each condition will have a single visit for data collection following routine clinical practice. No experimental treatments are given instead, the study gathers information during this one visit to assess disease characteristics and impact. During the visit, participants will complete questionnaires and clinical assessments specific to their condition. These include tools measuring symptom impact, hair loss severity, skin depigmentation, and quality of life related to each disease. This helps researchers better understand the real-world burden of these conditions. Participation involves only this one visit, with no long-term follow-up or additional procedures.

Researchers are evaluating the real-world effectiveness of deucravacitinib in adults diagnosed with moderate-to-severe plaque psoriasis. This observational study aims to understand how this treatment works outside of controlled clinical trials, focusing on health outcomes important to patients and doctors. It is sponsored by Bristol-Myers Squibb and conducted in France. Participants in this study have started treatment with deucravacitinib as prescribed by their doctors. The study observes these patients over time without altering their treatment plans. There are no placebo or comparison groups since this is an observational study that follows patients initiating deucravacitinib treatment. During the study, doctors will assess skin condition using the Physicians Global Assessment and measure quality of life with the Dermatology Life Quality Index at baseline and several points up to 24 months. Patients will also report symptoms like itch, skin pain, and fatigue using visual scales. Researchers will track how long patients remain on treatment and reasons for stopping. The study involves regular follow-ups at months 1, 4, 12, 18 optional, and 24 to gather this information.

Researchers are evaluating the safety and effects of a new medicine called NNC0487-0111 in people who have Heart Failure with preserved Ejection Fraction HFpEF or Heart Failure with mildly reduced Ejection Fraction HFmrEF and excess body weight. This phase 3 clinical trial aims to find out if NNC0487-0111 is safe and effective for treating these conditions compared to a placebo. Participants have HFpEF or HFmrEF and a body mass index of 30 or above. The study is sponsored by Novo Nordisk AS and uses a randomized, quadruple-masked design. Participants will receive either NNC0487-0111 or a matching placebo by injection under the skin once a week. The NNC0487-0111 is given in increasing doses over time. The study is parallel in design, meaning participants are randomly assigned to one of the two groups and receive that treatment throughout the trial. This treatment period extends for up to about 165 weeks. The study evaluates the time to certain heart failure events, hospitalizations, cardiovascular deaths, and other major cardiovascular events. During the study, participants will be monitored regularly to assess heart failure outcomes and kidney function, as well as quality of life using questionnaires like the Kansas City Cardiomyopathy Questionnaire. Safety and effectiveness are assessed through hospital visits, heart failure event tracking, and blood tests including kidney function and blood sugar levels. The total participation spans over three years, with ongoing evaluations to measure the time to heart failure events and cardiovascular outcomes. Participants receive close medical monitoring throughout the study period.

Researchers are evaluating the effectiveness of combining tarlatamab with durvalumab, carboplatin, and etoposide compared to the combination of durvalumab, carboplatin, and etoposide alone in treating patients with untreated extensive stage small-cell lung cancer ES-SCLC. The main goal is to see if the addition of tarlatamab can help patients live longer overall. This is a phase 3, open-label, randomized study conducted across multiple centers. Participants are assigned to one of two groups. One group receives tarlatamab combined with durvalumab, carboplatin, and etoposide for four treatment cycles, followed by maintenance treatment with tarlatamab and durvalumab. The other group receives durvalumab, carboplatin, and etoposide for four cycles, followed by durvalumab alone. All drugs are given as intravenous infusions. The study lasts up to approximately 3.5 to 4 years to assess survival and disease progression. During the study, participants will undergo regular assessments including evaluations of overall survival and progression-free survival. Other measures include response to treatment, disease control, duration of response, and monitoring for treatment-related adverse events. Blood tests will evaluate drug levels and antibody development. The research team will carefully monitor participants throughout the treatment and follow-up periods, keeping track of their health and any side effects.