Toulon

Researchers are studying patients with metastatic colorectal cancer (mCRC) who have a specific BRAFV600E mutation. This rare subtype of mCRC has poor prognosis and resistance to current treatments, especially in tumors with microsatellite stability or proficient mismatch repair. The study aims to collect detailed clinical data and biological samples to better understand treatment outcomes, resistance, and survival in real-world settings. Participants will provide blood samples and tumor tissue samples to support various research goals. The study will evaluate circulating tumor DNA during different lines of metastatic treatment to predict treatment response and resistance. It will also analyze the immune environment of BRAFV600E mCRC tumors and study specific subgroups with mismatch repair deficiencies. Clinical management data will be collected to inform future therapeutic approaches. During the study, patients will be monitored regularly with blood sample collections of 30 mL at each time point. Researchers will gather information about treatments, survival, and biological markers over time. The main outcome measured is overall survival from diagnosis up to five years. Patients must be able to comply with study procedures and provide informed consent. The study aims to improve knowledge of this aggressive cancer subtype and support development of new treatments.

Researchers are evaluating the safety and effectiveness of rilvegostomig compared to pembrolizumab, both combined with platinum-based doublet chemotherapy, as initial treatments for patients with metastatic non-squamous non-small cell lung cancer (mNSCLC) whose tumors express PD-L1. This Phase III, randomized, double-blind, global study focuses on patients whose tumors meet the PD-L1 expression threshold of 1% or higher and do not have certain genetic mutations or rearrangements that would require other targeted therapies. Participants receive either rilvegostomig or pembrolizumab intravenously on the first day of each 21-day treatment cycle. Both groups also receive platinum-based chemotherapy drugs such as carboplatin or cisplatin, administered intravenously up to four cycles, along with pemetrexed given intravenously on Day 1 of each cycle. The study monitors these treatments as first-line therapy for metastatic non-squamous NSCLC. During the study, participants undergo regular assessments including imaging scans to measure tumor size and response, as well as evaluations of organ and bone marrow function. Researchers track overall survival and progression-free survival for up to approximately five years. Safety is closely monitored throughout, and patients are followed long-term to assess outcomes related to treatment effectiveness and tolerability.

This is a Phase III, two-arm, randomized, double-blind, global, multicenter study assessing the efficacy and safety of rilvegostomig compared to pembrolizumab, both in combination with platinum-based doublet chemotherapy, as a first-line (1L) treatment for patients with squamous metastatic non-small cell lung cancer (mNSCLC) whose tumors express PD-L1 (tumor cells (TC) ≥ 1%).

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 studying patients with completely removed non-small cell lung cancer (NSCLC) who have common EGFR mutations (L858R and Del19). The study aims to include broad-panel centralized genetic testing at the start to better understand factors predicting outcomes and resistance to the drug osimertinib when used after surgery. It also investigates the molecular changes linked to cancer returning during or after osimertinib treatment to find better treatment options if the cancer comes back in a metastatic form. The study involves collecting plasma circulating tumor DNA (ctDNA) samples before surgery (optional), 4 to 8 weeks after surgery, before starting any adjuvant chemotherapy or osimertinib treatment, every six months during follow-up, and at relapse. Tumor tissue samples from surgery and optionally at relapse are also collected for molecular analysis. Patients may receive adjuvant chemotherapy if needed before starting osimertinib, which is given with the intent to treat for three years. Participants will be regularly followed every 3 to 6 months according to standard recommendations. Researchers will monitor genetic markers using blood and tissue samples to study cancer relapse and resistance. The main outcome is to assess the feasibility of this molecular monitoring approach over an 18-month period. Safety and long-term follow-up are included, aiming to improve treatment decisions for patients with resected NSCLC and EGFR mutations.

Researchers are studying patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM) in the United States and Europe to understand their characteristics, treatment patterns, and outcomes over time. The study focuses on individuals who are receiving mavacamten, other treatments for obstructive HCM, or no treatment due to intolerance or failure of prior therapies. The research includes a United States sub-study to evaluate mavacamten's safety and a European sub-study to assess both its effectiveness and safety in real-world settings. Participants may receive mavacamten according to its product label or other symptomatic therapies such as beta-blockers, non-dihydropyridine calcium channel blockers, or disopyramide based on standard care. The study includes those starting mavacamten, currently on other treatments, or untreated due to intolerance or failure of prior therapy. Treatment is observed during routine clinical care without altering prescribed therapy. Data collection occurs over several years to monitor long-term outcomes. During the study, participants will be regularly assessed for heart function and symptoms, including measuring the left ventricular outflow tract gradient and monitoring the incidence of new or worsening heart failure up to five years. Researchers will gather information on patient health, treatment safety, and heart function changes through echocardiography and symptom evaluations. The study allows for long-term observation to better understand real-world treatment effects and outcomes in obstructive HCM patients.

Researchers are conducting a global study to understand the impact of moderate to severe alopecia areata (AA), non-segmental vitiligo (NSV), and hidradenitis suppurativa (HS) on adolescents and adults. This study aims to assess the burden these conditions place on patients' quality of life and daily functioning in a large real-world population. The study involves participants diagnosed by a physician with one of the three conditions: AA, NSV, or HS. There are no interventional treatments or medications being tested in this study, as it is observational in nature. Data collection focuses on patient-reported outcomes and measures that evaluate disease severity and its effects. Participants will complete various questionnaires and assessments related to their condition, such as the Alopecia Areata Symptom Impact Scale (AASIS) for AA, the Severity of Alopecia Tool (SALT) for scalp hair loss in AA, the Facial Vitiligo Area Scoring Index (F-VASI) and Vitiligo Quality of Life Score (VitiQoL) for vitiligo, and the Dermatology Life Quality Index (DLQI) and International Hidradenitis Suppurativa Severity Scoring System (IHS4) for HS. These tools help researchers understand how symptoms affect quality of life and disease severity. The study collects information up to the day of the study visit.

This research aims to evaluate the real-world effectiveness of deucravacitinib treatment in adults diagnosed with moderate-to-severe plaque psoriasis. The study is conducted in France and focuses on understanding how this treatment performs outside of controlled clinical trial settings. Participants in this observational study will be newly starting deucravacitinib as prescribed by their treating clinician. There are no additional study treatments or placebo groups, as the study observes the outcomes of the treatment during routine clinical care. During the study, researchers will assess clinical outcomes including the Physician's Global Assessment (PGA) and the Dermatology Life Quality Index (DLQI) at baseline and at months 4, 12, 18 (optional), and 24. They will also monitor how long participants remain on deucravacitinib treatment, up to 24 months. These evaluations help to measure both the effectiveness and impact on quality of life for participants with plaque psoriasis.

Researchers are investigating whether ziltivekimab can treat people living with heart failure and inflammation. The study compares ziltivekimab, a new medicine not yet approved anywhere, to a placebo, an inactive substance that looks like the medicine but contains no active drug. Participants have an equal chance of receiving either treatment. The study is expected to last up to one year and four months and focuses on people with heart failure who also have systemic inflammation. Participants will receive either ziltivekimab or placebo by monthly injections under the skin. The doses are given once a month throughout the study period. The study lasts for 12 months of treatment following randomization, during which the effects of the medicine compared to placebo will be closely monitored. During the study, participants will undergo various assessments including a heart failure questionnaire called the Kansas City Cardiomyopathy Questionnaire (KCCQ) to measure symptoms and physical function over the 12 months. Other evaluations may include walking tests and heart function tests. Safety and health will be monitored regularly to understand how participants respond to the treatments and to track any side effects or changes in heart failure symptoms.

Researchers are evaluating ziltivekimab as a treatment for people living with heart failure and inflammation. This Phase 3 study compares ziltivekimab to a placebo in participants with heart failure who have mild to preserved ejection fraction and systemic inflammation. The study aims to assess the effect of ziltivekimab on cardiovascular death, heart failure hospitalization, or urgent heart failure visits over a period of up to 4 years. Participants will receive monthly injections of either ziltivekimab or a placebo using a pre-filled syringe or a pen-injector. The study medication is administered subcutaneously once a month for up to 4 years. The trial includes up to 20 clinic visits during which participants will be monitored and assessed. During the study, participants will use a study app on their phone to record all injections and complete questionnaires. Researchers will monitor participants for key outcomes like cardiovascular events and heart failure episodes from the time of randomization until the end of the study. Safety and health status will be regularly evaluated throughout the study period, which may last up to 48 months.