Poitiers

Researchers are investigating the drug bezuclastinib in an open-label, two-part Phase 2 study for patients with Advanced Systemic Mastocytosis (AdvSM), including Aggressive Systemic Mastocytosis (ASM), Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL). The study aims to evaluate the safety, effectiveness, and how the drug behaves in the body for these serious conditions. Bezuclastinib is given orally as tablets taken continuously in 28-day cycles. The study has two parts: Part I focuses on identifying safe and tolerable doses of bezuclastinib over 18 months, while Part II evaluates its effectiveness by measuring the objective response rate and confirming the relationship between drug exposure and response during another 18-month period. Participants will undergo assessments including clinical evaluations, laboratory tests, and monitoring of their disease status to determine treatment effects and safety. Researchers will track the drug's impact on the disease and patient health throughout the study, which involves continuous treatment and follow-up over the specified time frames.

Researchers are evaluating the safety and effectiveness of elenestinib (BLU-263) combined with symptom-directed therapy (SDT) compared to placebo plus SDT in people with indolent systemic mastocytosis (ISM) whose symptoms are not well controlled by SDT alone. This Phase 2/3 randomized, double-blind, placebo-controlled study includes participants with ISM and smoldering systemic mastocytosis, and also involves groups for pharmacokinetic studies and participants who previously received a selective KIT inhibitor. The study is divided into multiple parts. Parts 1 and 2 enroll participants with ISM who will receive either elenestinib oral tablets or placebo alongside their symptom-directed therapy. Participants from Part 2 may continue into Part 3, which is an open-label extension where all receive elenestinib. Part K enrolls participants with ISM who have prior experience with selective KIT inhibitors. The study tracks treatment effects and safety over time. Participants will be monitored for up to 5 years, with assessments including the number of treatment-emergent adverse events, changes in symptom scores measured by the ISM-Symptom in Assessment Form, and overall safety monitoring. Evaluations occur at baseline, 13 weeks, 49 weeks, and throughout the long-term follow-up. The study also includes detailed tracking of symptom control and adverse events to evaluate the impact of treatment on participants' health and quality of life.

Researchers are evaluating sotatercept as a potential treatment for pulmonary arterial hypertension (PAH), a condition where blood vessels in the lungs thicken and narrow, causing high blood pressure in the lungs and overworking the heart. PAH symptoms include difficulty breathing and reduced ability to be active. Current standard treatments address symptoms but do not stop disease progression. This Phase 3 study focuses on the long-term safety and tolerability of sotatercept when added to standard PAH therapy. Participants in this long-term follow-up study receive sotatercept through subcutaneous injections every three weeks. Only individuals who completed prior sotatercept PAH studies without early discontinuation may join. This study continues the observation and assessment of participants over an extended period to learn about the effects and safety of sotatercept combined with background PAH treatments. During the study, participants will be regularly monitored for adverse events, treatment discontinuations, and the presence of anti-drug antibodies for up to approximately 90 months. Laboratory tests will evaluate blood components such as platelets, hemoglobin, creatinine, bilirubin, and liver enzymes. Changes from baseline in body weight, blood pressure, and electrocardiogram readings will also be tracked. The study involves adherence to visit schedules and compliance with study procedures to ensure comprehensive long-term safety data collection.

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 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 studying adults with confirmed Primary Biliary Cholangitis (PBC) and cirrhosis, a scarring of the liver caused by damage to bile ducts. PBC is a slowly progressing disease that causes bile acid buildup and further liver damage, which can lead to cirrhosis. This study aims to evaluate if elafibranor, a daily medication, can prevent worsening clinical outcomes such as the need for liver transplant or death, compared to a placebo. It also looks at the safety of long-term elafibranor use and its effect on symptoms like itching and tiredness. Participants will take either an 80 mg tablet of elafibranor or a matching placebo once daily for up to 3.5 years in a double-blind setup, meaning neither the participants nor researchers know who receives which treatment. This long-term treatment period is designed to monitor the drug's impact over time. The study includes two groups: one receiving elafibranor and the other receiving placebo, with treatment lasting up to approximately 42 months. During the study, participants will be regularly assessed from the start until 4 weeks after treatment ends, with a maximum involvement of 3.5 years. Researchers will measure event-free survival, tracking if participants avoid clinical events indicating disease worsening. Safety monitoring will include tracking side effects and overall health, while symptom impact will be evaluated. Participants will provide informed consent and follow the study protocol throughout this extended observation period.

Researchers are evaluating the effects of pelacarsen (TQJ230), given as a monthly injection under the skin, in people with mild to moderate calcific aortic valve stenosis. This study aims to see if pelacarsen can safely slow the progression of this heart valve condition compared to a placebo. The trial is a phase 2, randomized, double-blind, placebo-controlled study conducted at multiple centers. Participants will receive either pelacarsen 80 mg or a matching placebo once a month. Before starting the treatment, they must have elevated lipoprotein(a) levels and be optimally treated for existing cardiovascular risk factors. The study focuses on those aged 50 to under 80 years with mild or moderate calcific aortic valve stenosis. During the 36 months of participation, researchers will monitor changes in peak aortic jet velocity and aortic valve calcium score to assess disease progression. Safety, tolerability, and the impact of the treatment will be evaluated. Participants will undergo regular assessments, including laboratory tests and clinical evaluations, to track heart valve condition and overall health throughout the study.

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 evaluating CRB-701, an antibody-drug conjugate targeting nectin-4, in adults with advanced solid tumors that express nectin-4. This Phase 1/2 clinical trial aims to find a safe and effective dose of CRB-701 and to determine which cancers might respond to this treatment. The study includes three parts focusing on safety, dosing, and effectiveness, especially for tumors resistant to other treatments or lacking standard therapies. The study is divided into three parts. Part A involves escalating doses of CRB-701 alone to identify the maximum tolerated dose and recommended dose for further study. Part B tests two dose levels of CRB-701 alone and combined with an anti-PD-1 checkpoint inhibitor to optimize dosing. Part C further explores CRB-701 alone or with anti-PD-1 in up to seven groups of participants. Treatments are given by intravenous infusion. Participants will attend clinic visits for infusions and undergo various tests including blood work, CT or MRI scans, and other assessments to monitor tumor response and safety. The main outcomes measured are dose safety, tolerability, and objective tumor response over periods ranging from 21 days to up to 6 months. Ongoing monitoring includes safety assessments and evaluation of treatment effects over time.