Poitiers

Researchers are investigating CGT9486, also known as bezuclastinib, in an open-label Phase 2 study for patients with Advanced Systemic Mastocytosis AdvSM. This includes those diagnosed with 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, pharmacokinetics, and pharmacodynamics of bezuclastinib in this patient population. Participants will receive bezuclastinib tablets orally, taken continuously in 28-day cycles. The study is divided into two parts Part I focuses on identifying effective and tolerable dosing exposures over 18 months, while Part II evaluates the drugs efficacy by measuring objective response rates and confirming the exposure-response relationship, also over 18 months. Additional assessments include effects on mutation allele burden, serum tryptase levels, histopathologic changes, spleen and liver volume, and safety monitoring. During the study, participants will undergo various clinical evaluations, including laboratory tests, imaging to monitor organ size changes, and assessments of disease response and progression. Researchers will track adverse events and pharmacokinetic profiles throughout the 18 months. The study involves continuous monitoring of participants to understand the treatments impact on survival and disease progression over this period.

Researchers are evaluating the safety and effectiveness of elenestinib BLU-263 combined with symptom directed therapy SDT compared to placebo plus SDT in adults with indolent systemic mastocytosis ISM whose symptoms are not well controlled by SDT alone. This randomized, double-blind, placebo-controlled Phase 23 study includes multiple parts to assess different doses and durations of elenestinib treatment, including an open-label extension for participants finishing earlier phases. The study also enrolls participants who have previously received an approved selective KIT inhibitor and includes pharmacokinetic groups. Participants receive oral elenestinib or placebo once daily alongside SDT, which is personalized based on individual symptom management needs. Part 1 focuses on short-term treatment lasting up to 12 weeks, while Part 2 extends treatment to approximately 48 weeks. Part 3 and other parts allow treatment for up to about 5 years. The study monitors participants through these phases to evaluate how elenestinib affects symptoms, disease markers, and safety over time. During the study, participants undergo regular assessments of symptoms using the ISM-Symptom Assessment Form ISM-SAF, laboratory tests including serum tryptase and KIT D816V allele levels, bone marrow evaluations, and quality of life measures. Researchers track adverse events and changes in disease-related factors at various points up to 5 years. This thorough monitoring helps measure treatment effects and safety over both short and long-term periods, with total participation lasting several years depending on the study part.

Researchers are studying pulmonary arterial hypertension PAH, a condition where lung blood vessels become thick and narrow, causing high blood pressure in the lungs and making it hard for the heart to work. PAH can cause difficulty breathing and limit activity. While standard treatments help symptoms, they do not stop the disease from worsening. This research focuses on sotatercept, a targeted therapy aimed at specific proteins involved in PAH, to learn about its long-term safety and tolerability when added to usual PAH treatments. Participants in this long-term follow-up study, who previously took part in certain sotatercept trials, may continue receiving sotatercept by subcutaneous injection every three weeks. Those coming from blinded studies start at 0.3 mgkg with possible increases up to 0.7 mgkg, while those from unblinded studies continue their current dose with possible titration to 0.7 mgkg. The study monitors participants over an extended period to assess continued effects alongside their usual PAH therapy. During the study, participants will have regular assessments including monitoring for adverse events, blood tests for blood components and chemistry, body weight, blood pressure, and ECG readings. Researchers will also evaluate exercise capacity, heart function markers, and risk scores related to PAH. The study aims to follow participants for up to approximately 7 to 8 years to understand long-term safety, treatment tolerability, and health changes while using sotatercept with standard PAH care.

Researchers are evaluating the effects of enicepatide, a dual GLP-1GIP receptor agonist, at multiple doses compared with placebo for weight management in adults with obesity or overweight who do not have Type 2 diabetes. This Phase III, randomized, double-blind study aims to assess both the efficacy and safety of once-weekly enicepatide in this population, addressing weight-related comorbidities such as prediabetes, hypertension, and cardiovascular conditions. Participants will be randomly assigned to receive either placebo or one of three enicepatide dosing regimens, administered once weekly via an integrated drug-device combination product. The treatment phase lasts through 72 weeks, during which changes in body weight and other health measures are monitored. The study includes multiple assessments to track body weight percentage change, waist circumference, fasting glucose and insulin levels, lipid profiles, blood pressure, and quality of life measures. Throughout the study, participants will undergo regular evaluations including physical examinations, laboratory tests, and questionnaires related to physical functioning and urinary incontinence. Researchers will monitor adverse events, patient-reported health questionnaires, and biomarkers at baseline and weekly intervals through week 72. This long-term follow-up allows for a comprehensive assessment of treatment effects and safety in participants managing obesity or overweight without Type 2 diabetes.

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.

This research aims to describe the clinical, histological, and radiological features of rare primary liver cancers. It focuses on collecting tumor and blood samples to better understand these cancers and to evaluate how well treatments used in real-world practice work, with the goal of identifying the best treatment sequences. The study serves as a foundation for future research to find new molecular and imaging biomarkers that could improve diagnosis and prognosis. The study is observational and retrospective, meaning it reviews past cases from multiple centers in France. It collects biological samples and clinical data from patients diagnosed with rare primary liver cancers after January 2018. The study evaluates treatments patients have received in clinical practice without assigning any new treatments or interventions. Participants data, including clinical characteristics, tumor biology, and imaging, will be reviewed for up to five years from diagnosis. Researchers will measure outcomes such as recurrence-free survival for patients without metastases, progression-free survival for those with metastases, and overall survival. The study includes both living patients who consent to participate and deceased patients, aiming to gather comprehensive information to support future translational studies.

This research aims to evaluate elafibranor, a study drug, compared to a placebo in adults with Primary Biliary Cholangitis PBC and cirrhosis, a liver disease causing bile duct damage and scarring. The trial focuses on whether elafibranor can better prevent worsening of the disease, including progression leading to liver transplant or death, and also assesses long-term safety and symptom impact such as itching and tiredness. Participants will be randomly assigned to take either an 80 mg tablet of elafibranor or a matching placebo tablet once daily, orally, with or without food. This double-blind treatment period can last up to 3.5 years for each participant, with tablets taken at approximately the same time each morning. The study is designed to compare these two groups over the long term. During the study, participants will undergo regular assessments including physical exams, vital signs, electrocardiograms, laboratory tests, and symptom questionnaires at intervals up to 3.5 years. Researchers will measure liver function tests, symptom scales, liver stiffness, and clinical outcomes related to disease progression. Safety is monitored through adverse event tracking and laboratory parameters, with follow-up extending to four weeks after the last dose. Overall participation may last up to 3.5 years.

This trial studies adults aged 50 to under 80 with mild or moderate calcific aortic valve stenosis and elevated lipoproteina levels. Researchers are assessing the safety, tolerability, and ability of pelacarsen TQJ230 given once monthly by injection to slow the progression of this heart valve condition. The study compares pelacarsen to a placebo in a randomized, double-blind design. Participants receive either pelacarsen 80 mg or a matching placebo as a subcutaneous injection monthly. They continue treatment and monitoring for up to 36 months to observe changes in heart valve narrowing and calcium buildup. The study also tracks lipoproteina levels and clinical heart-related events during this period. Throughout the study, participants will have regular assessments including imaging to measure aortic valve function and calcium score, blood tests for lipoproteina, and monitoring for safety. The main outcomes analyzed after 36 months include changes in valve jet velocity and calcium score, alongside clinical events. Participants remain under medical care while being observed for any effects of the study drug or placebo.

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.

Malignant hypertension is a very serious form of high blood pressure that can be fatal if untreated. This research aims to create the first large, multicenter database to better understand this disease, including its modern epidemiology, how patients are currently managed, and the diseases diagnostic criteria. The study will help improve knowledge and may lead to new treatment trials and evidence-based recommendations. The study is an observational registry enrolling patients diagnosed with malignant hypertension based on classic definitions, including severe blood pressure elevation and organ damage. It plans to recruit 500 patients and follow them for five years to study their prognosis and the impact of different patient characteristics and organ involvement. By collecting detailed data on disease features and care pathways, the study hopes to update definitions and management approaches. Participants will be observed over five years, with researchers collecting information on their health status, organ damage, and treatment. The main outcome measured is the five-year prognosis of patients. This long-term follow-up will provide detailed knowledge about the disease course and help identify factors influencing outcomes, without any study treatments or interventions being assigned.