Pau

Researchers are conducting a French multicenter retrospective study to describe the clinical, histological, and radiological features of rare primary liver cancers. The study aims to collect biological tumor and blood samples and evaluate the effectiveness of treatments used in clinical practice to determine the best therapeutic sequences. This research will serve as the foundation for future translational studies to identify new molecular, histological, circulating, and radiological tumor biomarkers useful for diagnosis, prognosis, and treatment guidance. This study involves collecting data from patients diagnosed with rare liver cancers such as hepatocholangiocarcinoma, fibrolamellar hepatocellular carcinoma, epithelioid hemangioendothelioma, and hepatic angiosarcoma since January 1, 2018. Both living patients who agree to participate and deceased patients are included. Biological samples and tumor blocks are collected for analysis. Treatments received by patients in routine practice are reviewed to assess their efficacy and help define optimal treatment sequences. Participants provide consent for biological studies if living, and their medical records and tumor characteristics are reviewed. Researchers will describe the clinical, histological, and radiological tumor features and monitor outcomes up to five years from diagnosis. This detailed data collection supports long-term evaluation of rare liver cancers and aids in developing future biomarkers and therapeutic strategies.

Researchers are evaluating the efficacy and safety of rilvegostomig compared to pembrolizumab as first-line treatments for patients with metastatic non-small cell lung cancer (mNSCLC) whose tumors have high PD-L1 expression. This Phase III, randomized, double-blind, and global study focuses on participants with stage IV mNSCLC who do not have certain genetic mutations or rearrangements and are eligible for systemic therapy. Participants receive either rilvegostomig or pembrolizumab intravenously on Day 1 of each 21-day cycle. The study compares these two biological treatments given as monotherapy. Both groups will be monitored over time to assess treatment impact and safety. Throughout the study, participants undergo evaluations including tumor measurements by CT or MRI, performance status assessments, and organ function tests. Researchers will measure overall survival and progression-free survival for up to approximately five years. Tumor samples are collected before treatment for central testing, and participants’ health and treatment responses are closely followed during the trial period.

Researchers are evaluating the safety and effectiveness of combining durvalumab and domvanalimab compared to durvalumab plus placebo in adults with locally advanced (Stage III), unresectable non-small cell lung cancer (NSCLC) whose disease has not worsened after definitive platinum-based concurrent chemoradiation therapy. This Phase III, randomized, double-blind, placebo-controlled, international study involves multiple centers. Participants receive intravenous infusions of durvalumab and domvanalimab or durvalumab and placebo. The treatments are given after patients have completed concurrent platinum-based chemotherapy and radiation therapy with a total radiation dose of approximately 60 Gy. The study monitors patients over time to assess treatment effects and safety. During the study, participants undergo evaluations including tumor tissue analysis for PD-L1 status, performance status assessments, and monitoring of organ and marrow function. The main outcome measured is progression-free survival up to 8 years after randomization. Researchers also monitor for any adverse effects and disease progression throughout the study period.

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.

Malignant hypertension is a very severe type of high blood pressure that can be fatal if not treated. It mainly affects younger adults aged 35 to 55 and carries a high risk of serious heart and kidney problems. Despite its severity and increasing cases, research on malignant hypertension is limited, with diagnostic criteria and treatment guidelines that have not changed since 1929. This study aims to create the first prospective, multicenter registry to better understand the disease's epidemiology, care practices, and biological aspects, and to modernize its definition and diagnosis. The study plans to enroll 500 patients diagnosed with malignant hypertension based on classic criteria, including severe high blood pressure above 180/110 mmHg and evidence of organ damage. It will collect detailed data on patient characteristics, affected organs, and treatment approaches used in various centers. This registry will help develop new diagnostic and treatment recommendations based on solid scientific evidence and may lead to future therapeutic trials. Participants will be followed to evaluate their health outcomes over five years, focusing on their cardiovascular and renal prognosis. Researchers will analyze how patient profiles and the number and type of organ damage affect their long-term outlook. The study will document epidemiology, care pathways, organ involvement, and management strategies in detail to improve understanding and care of malignant hypertension.

Researchers are studying metastatic renal cell carcinoma (RCC), a type of kidney cancer that spreads to other parts of the body, affecting many patients annually in France. This study focuses on patients with oligoprogressive disease, where only a few metastatic sites (1 to 3) show progression while the rest remain controlled under ongoing systemic treatments like targeted therapies or immunotherapy. The goal is to evaluate stereotactic radiotherapy (SRT) as a focused treatment to control these progressing sites and potentially delay the need for changing systemic therapies. The study involves delivering stereotactic radiotherapy, which uses high doses of radiation in one or a few sessions to target metastatic sites specifically. Patients with up to three progressive metastases eligible for SRT will receive this treatment concurrently or sequentially alongside their current systemic therapy. This approach aims to control tumor growth locally and possibly stimulate a broader immune response. The trial is a Phase II study, assessing this treatment strategy in patients receiving first or second-line systemic therapies. Participants will undergo imaging scans to confirm disease progression and lesion sizes, with follow-up assessments to monitor progression-free survival six months after randomization. Researchers will evaluate how well the targeted radiotherapy controls tumor sites and delays further disease progression. Patients will be closely monitored for treatment effects, ability to continue systemic therapy, and overall safety throughout the study period.

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 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.

Researchers are evaluating how well elacestrant works compared to standard endocrine therapy in adults with node-positive, Estrogen Receptor-positive (ER+), Human Epidermal Growth Factor-2 negative (HER2-) early breast cancer who are at high risk of the cancer returning. This is a Phase 3 global, multicenter, randomized, open-label study focusing on participants who have had early invasive breast cancer removed and meet specific receptor and risk criteria. The study aims to understand which treatment better prevents invasive breast cancer over up to five years. Participants will receive either elacestrant or one of several standard endocrine therapies, including anastrozole, letrozole, exemestane, or tamoxifen, all given as oral tablets. Treatments will be administered according to the study plan, with careful monitoring throughout the trial. The study includes adults who have already received between 24 and 60 months of prior endocrine therapy, with or without certain inhibitors, and who have completed or stopped these treatments as required. During the study, participants will be monitored for invasive breast cancer-free survival for up to five years. Researchers will perform regular assessments to track treatment effects, side effects, and cancer recurrence. The study also includes safety monitoring and may involve additional tests or evaluations as needed to ensure participant well-being throughout the trial.

Researchers are investigating the effects of teplizumab compared with a placebo in children and young adults aged 1 to 25 years who have recently been diagnosed with Stage 3 type 1 diabetes (T1D). This Phase 3, multicenter, randomized, double-blind, placebo-controlled study aims to assess changes in blood sugar control and dependence on prandial insulin over one year. The study follows established criteria for Stage 3 T1D diagnosis and includes participants on standard insulin therapy. Participants will receive either teplizumab or placebo through intravenous infusion. Both treatments are given as a solution for injection. The study spans approximately 84 weeks (18 months) for each participant, with the main treatment period lasting 52 weeks. The trial compares the effects of the two treatments on diabetes management and insulin use over this time. During the study, participants will undergo evaluations of their blood sugar control, including measurements of glycated hemoglobin (HbA1c) and insulin use. For those in European Union countries aged 5 years and older, additional tests will measure insulin production response after a meal. Safety and health will be monitored through regular clinical assessments, lab tests, and antibody screening. The study tracks changes from the beginning through the 52-week treatment period to understand the impact of teplizumab on diabetes progression.