Bourdeaux

Researchers are evaluating the efficacy and safety of empasiprubart in adults with Chronic Inflammatory Demyelinating Polyneuropathy CIDP. This Phase 3, randomized, double-blinded, placebo-controlled study compares empasiprubart to placebo to better understand its impact on CIDP symptoms and disease progression. The study has two parts Part A lasts 24 weeks 6 months, where participants receive either empasiprubart or placebo via intravenous infusion. After Part A, all participants enter Part B for 96 weeks 24 months during which everyone receives empasiprubart. Participants who received empasiprubart in Part A will receive a placebo dose once during Part B to maintain the study blind. Participants will have regular assessments including measurements of disability using the adjusted inflammatory neuropathy cause and treatment aINCAT score, grip strength, and other neurological and quality of life scales. Safety is monitored throughout the study. The total participation period spans up to 120 weeks, with evaluations at multiple time points to track changes from baseline and any adverse events.

Researchers are studying clinical outcomes in patients undergoing transcatheter mitral valve replacement TMVR for mitral insufficiency. This multinational registry focuses on patients treated with TMVR in real-world practice and collects data on those who have undergone screening for TMVR. Some historical data include patients who received other treatments like edge-to-edge repair, mitral valve surgery, or medical therapy. The registry includes patients who successfully underwent TMVR with any device. Patients who failed TMVR screening and underwent other treatments such as mitral valve edge-to-edge repair, mitral valve surgery, or medical therapy were previously included but are no longer systematically enrolled. The study collects data over various timepoints to observe the effects of these therapies. Participants are followed for at least 30 days after treatment with echocardiography and clinical evaluations. Researchers measure mitral insufficiency severity, device-related complications within 30 days, and longer-term outcomes like mortality, rehospitalization for heart failure, and unplanned mitral valve interventions over 12 months. This observational registry aims to understand outcomes and safety in patients treated with TMVR and related therapies in routine care.

This research trial investigates the treatment of well to moderately differentiated neuroendocrine tumors of the lung and thymus. It compares two therapies 177Lu-edotreotide, a type of peptide receptor radionuclide therapy, and everolimus, an oral drug. The study focuses on patients whose tumors have high expression of somatostatin receptors as confirmed by imaging and aims to determine which treatment better delays disease progression. Participants will be randomly assigned in a 32 ratio to receive either 177Lu-edotreotide or everolimus. The 177Lu-edotreotide group will receive 6 cycles of treatment spaced between 6 to 8 weeks apart, while the everolimus group will take a daily oral dose of 10 mg until disease progression, intolerable side effects, or death. Treatment will continue unless stopped due to progression, toxicity, or patient choice. Throughout the study, patients will undergo regular tumor assessments using MRI or CT scans every 12 weeks to monitor disease progression. Researchers will measure progression-free survival as the primary outcome over about three years per participant. Other assessments include tumor response rates, overall survival, and quality of life questionnaires. Safety and long-term follow-up will continue even if treatment stops, unless participants withdraw consent entirely.

Researchers are evaluating an artificial intelligence AI system designed to improve the prediction of outcomes for patients undergoing transcatheter heart valve interventions for conditions affecting the aortic, mitral, and tricuspid valves. This non-interventional, retrospective study analyzes data from multiple specialized centers worldwide to validate AI algorithms that automatically analyze cardiac imaging and clinical data. The goal is to enhance patient selection, intervention planning, and outcome predictions while reducing human error and variability in image interpretation. The study collects medical imaging data, including multi-slice cardiac computed tomography CT and transesophageal echocardiography TEE, along with preoperative clinical information from patients who have undergone various heart valve procedures. These include transcatheter aortic valve implantation TAVI, transcatheter mitral valve implantation TMVI, transcatheter tricuspid valve intervention TTVI, and edge-to-edge repair procedures for the mitral M-TEER and tricuspid valves T-TEER using specific device generations. The AI framework applies deep learning, including convolutional neural networks, to segment anatomical structures and measure them accurately from images. Participants data are analyzed retrospectively to compare AI-generated automated measurements with manual assessments and to evaluate the accuracy of AI predictions against actual patient outcomes at 30 days post-procedure. The study also monitors the performance of AI algorithms throughout an average of two years of retrospective data collection. This process aims to confirm the AIs ability to support clinical decision-making and improve intervention results in heart valve disease patients.

This research focuses on people with aging psychiatric disorders who face cognitive decline affecting perception, memory, attention, and problem-solving. It aims to evaluate the Perceive, Recall, Plan and Perform PRPP task analysis system, a tool used by occupational therapists to assess cognitive difficulties and support decisions about maintaining independent living or finding suitable housing. The study is conducted in a specialized unit addressing disability and loss of autonomy in psychiatric patients. Participants undergo a structured program starting with assessments and PRPP evaluations of two activities over the first three months. Based on these results, a referral decision is made regarding the participants living situation. From three to nine months, participants receive personalized outpatient psychosocial rehabilitation and occupational therapy interventions guided by the PRPP. At nine months, a reassessment with PRPP and questionnaires helps confirm the care and living orientation plan. During the study, participants will be evaluated regularly by a mobile multi-professional team and through questionnaires completed by care providers and psychiatrists. The main outcome measured is the efficiency of the PRPP system in guiding living orientation decisions after nine months. Secondary outcomes include tracking hospitalizations within twelve months. The study involves close monitoring of participants cognitive and functional abilities while supporting their autonomy in daily life.

Calciphylaxis, also called uremic calcifying arteriolopathy UCA, is a rare condition causing painful skin lesions due to small blood vessel calcification and clotting. This disease mainly affects patients with end-stage renal disease ESRD who require hemodialysis. Researchers are evaluating the safety and effectiveness of adding rheopheresis, a special blood filtration treatment, to the standard care for calciphylaxis in these patients through a prospective randomized controlled trial. In this study, participants will be randomly assigned to one of two groups. The experimental group will receive rheopheresis in addition to standard care, involving an induction phase of 3 sessions in the first week followed by 2 sessions weekly for 3 weeks, then a maintenance phase with 1 session per week up to week 11. The comparator group will receive sham-apheresis sessions on the same schedule, which mimics the procedure without actual filtration. Rheopheresis uses a machine to remove certain high molecular weight proteins from plasma to help treat microcirculation problems. Participants will be followed for 12 weeks during treatment with regular assessments of wound healing, pain levels, quality of life, antibiotic usage, hospital discharge days, survival, and inflammatory protein changes. The primary outcome is the percentage of patients achieving complete healing of calciphylaxis lesions after 12 weeks. Secondary outcomes include partial healing, new lesion occurrence, pain and analgesic use, and overall survival at 12 weeks and one year. Safety and efficacy data will help determine the added value of rheopheresis in calciphylaxis care.

Researchers are evaluating XmAb819, a monoclonal bispecific antibody, in adults with relapsed or refractory clear cell renal cell carcinoma ccRCC and other solid tumors who have progressed after standard therapy. This Phase 1, multicenter, open-label study aims to assess the safety and tolerability of XmAb819, determine the minimum safe and biologically active dose, and establish a recommended dose for further research. The study also explores pharmacokinetics, pharmacodynamics, immunogenicity, and preliminary antitumor activity. The study has two parts dose escalation and dose expansion. During dose escalation, dosing schedules for intravenous IV and subcutaneous SC administration of XmAb819 are established, including priming and step-up priming doses. In the dose expansion phase, participants may receive XmAb819 by IV or SC routes. Treatments are given in multiple doses to assess safety and biological activity. Participants will be evaluated through assessments of adverse events, dose-limiting toxicities, and pharmacokinetic measures such as maximum concentration and area under the curve. Tumor response and progression-free survival will be monitored. Safety and tolerability will be followed for 28 days after dosing, with additional outcome measures assessed up to 56 days or 42 days depending on the endpoint. The study lasts until completion in December 2028.

Researchers are evaluating the safety and performance of the Occlutech Atrial Flow Regulator device in patients with heart failure. This international, multicenter follow-up registry aims to identify unknown side effects and determine when this treatment is appropriate. The study focuses on patients with chronic symptomatic heart failure and monitors outcomes over a 36-month period after device implantation. Participants will receive the Occlutech Atrial Flow Regulator, which is an interatrial shunt device delivered through a catheter. Treatments and procedures follow clinical routine and device instructions, performed by experienced physicians at equipped centers. The study tracks device performance and cardiovascular events at 12, 24, and 36 months after implantation. During the study, participants will undergo assessments including vital signs, lab tests, quality of life questionnaires, ECGs, and echocardiograms. Researchers will measure major adverse cardiovascular and neurological events within one year, as well as heart function, exercise capacity, and medication changes over three years. Safety and efficacy will be closely monitored throughout the follow-up period.