Nancy

Researchers are evaluating the effects of early apneic ventilation compared to usual care with ultra-protective lung ventilation in patients with severe acute respiratory distress syndrome (ARDS) who require venovenous extracorporeal membrane oxygenation (ECMO). This Phase 3, open-label, multicenter trial aims to examine whether early apneic ventilation can reduce lung injury, shorten ECMO duration, and lower mortality at 60 days in this critically ill population. Participants are randomized to receive either ECMO plus near apneic ventilation or ECMO plus ultra-protective lung ventilation. Near apneic ventilation is applied during the first three days of ECMO using BIPAP/APRV or pressure-controlled ventilation, with specific settings to maintain airway pressures and low respiratory rates. After three days, apneic ventilation may continue or standard ultra-protective ventilation is used. The ultra-protective lung ventilation group receives low tidal volume and pressure ventilation settings until ECMO weaning. Prone positioning during ECMO is allowed at the physician's discretion in both groups. Throughout the study, researchers monitor mortality, need for lung transplantation, ongoing ECMO support, and days alive without ECMO up to day 60. Participants undergo clinical assessments and ventilator management according to the assigned strategy. Consent procedures accommodate emergency inclusion with surrogate consent when needed, and follow-up includes evaluation of lung recovery and survival outcomes over the 60-day period.

Researchers are studying advanced renal cell carcinoma (RCC) that has returned after prior adjuvant therapy. The trial aims to find out if treatment with belzutifan and zanzalintinib helps patients live longer and delays disease progression compared to treatment with cabozantinib. This is a Phase 3 randomized study focusing on participants with recurrent advanced RCC who have previously received anti-PD-1/L1 therapy. Participants are randomly assigned to receive one of two oral drug regimens: either belzutifan combined with zanzalintinib, both taken once daily, or cabozantinib alone, also taken once daily. The study compares these treatments to assess their effects on disease control and overall survival. During the study, participants will be monitored for progression-free survival and overall survival for up to approximately 73 months. Researchers will evaluate how well the cancer responds to treatment and track any changes in health status over time. Safety and effectiveness of the treatments will be closely followed throughout the study period.

Food Protein Induced Enterocolitis Syndrome (FPIES) is a non-IgE mediated food allergy that mainly appears in infancy and can cause severe vomiting, diarrhea, and dehydration. This condition is often unfamiliar to many clinicians, and its diagnosis relies on specific clinical criteria established in 2017. This study aims to collect detailed clinical information and allergy test results for children diagnosed with acute FPIES and to describe its progression and atypical forms over three years in a French population. The study will include children aged 0 to 17 years with confirmed acute FPIES, diagnosed either by the 2017 JACI clinical criteria or by oral food challenge. Allergy tests such as oral food challenges, skin prick tests, and IgE blood tests will be used to monitor patients. Participants will be seen by allergologists at the start and annually for three years. If tolerance to the offending food is not acquired, an oral food challenge will be conducted in a hospital setting to evaluate resolution. This multicenter French prospective study will gather data on FPIES evolution, including the development of IgE sensitization and clinical symptoms of IgE-mediated allergy. Throughout the study, children will undergo clinical evaluations and allergy testing at inclusion and yearly follow-up visits. Researchers will assess tolerance acquisition over an average of six years, monitor multiple FPIES cases, and record personal atopic conditions. The study will last three years for inclusion and three years of follow-up per patient, aiming to improve management of FPIES in France by understanding its unique food triggers and long-term outcomes.

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 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 evaluating the effectiveness and safety of BHV-7000 in adults with refractory focal onset epilepsy, a condition where seizures originate in one area of the brain and do not respond well to current treatments. This Phase 2/3 clinical trial aims to determine whether BHV-7000 can reduce seizure frequency in this population. The study is divided into two parts. In Part A, participants are randomly assigned to receive either 25 mg or 50 mg of BHV-7000, or a matching placebo, taken once daily. After completing Part A, participants move to Part B, where they are randomized to receive 75 mg of BHV-7000 or a matching placebo, also taken once daily. Both parts are randomized and double-blinded to ensure unbiased results. Participants will be monitored from Week 8 to Week 20 of each part for changes in average seizure frequency, serious adverse events, discontinuations due to side effects, and laboratory abnormalities. Researchers will track seizure diaries and assess safety and tolerability throughout the study. The total duration includes both study parts with regular evaluations to measure the drug’s impact and participant safety.

Researchers are evaluating the effectiveness of axatilimab combined with corticosteroids compared to a placebo plus corticosteroids as the initial treatment for moderate or severe chronic graft-versus-host disease (cGVHD). This Phase 3, randomized, double-blind, placebo-controlled study focuses on patients aged 12 years and older who have new-onset moderate or severe cGVHD following an allogeneic hematopoietic cell transplant (allo-HCT). Participants will receive either axatilimab (INCA034176) or a placebo, both given by intravenous infusion, along with corticosteroids administered orally or by IV infusion. The study will monitor how well these treatments control the disease when started as the first systemic therapy. Treatment schedules and dosing details will be managed as per the study protocol during the trial period. Throughout the study, participants will be closely monitored for event-free survival (EFS) for up to three years. Researchers will assess the safety and effectiveness of the therapies, track disease progression, and evaluate any side effects. Participants will undergo regular clinical evaluations and laboratory tests as part of the study follow-up procedures.

Ulcerative Colitis (UC) is a long-lasting inflammatory bowel disease affecting the colon, mainly impacting adults between 30 and 40 years old. This condition causes recurring inflammation that starts in the rectum and can spread through the colon, leading to disability and reduced quality of life. The study aims to identify new biomarkers in blood, stool, and tissue that can predict how patients will respond to various targeted treatments, enabling more personalized therapy choices for people with moderate to severe UC. The study involves collecting samples through procedures like endoscopic biopsies, blood draws, and stool sampling from patients receiving different biologic or small molecule therapies such as anti-TNF agents, anti-IL12/23 agents, anti-integrins, and JAK inhibitors. These treatments are prescribed based on current medical guidelines and the patients' disease status. Patients must also undergo endoscopy to assess disease activity as part of standard care. Participants will be monitored over time to evaluate their clinical response, deep clinical remission, mucosal healing, and symptomatic remission at multiple time points including weeks 14, 26, and 52. Researchers will use these measures to assess treatment effectiveness and the value of identified biomarkers. The study also includes safety monitoring and requires participants to comply with study procedures and provide informed consent.

Researchers are evaluating the effectiveness and safety of KarXT combined with KarX-EC for treating cognitive impairment in people with mild to moderate Alzheimer's Disease. This Phase 3 study focuses on individuals aged 60 to 85 who have a confirmed diagnosis of Alzheimer's disease according to updated clinical criteria and have specific cognitive scores indicating mild to moderate dementia. Participants will receive either the study drugs KarXT and KarX-EC or a placebo, each given at specified doses on certain days. The study is randomized, double-blind, and placebo-controlled to compare the effects of these treatments on cognitive function. Those already taking certain Alzheimer's medications must have stable doses before and during the study. During the study, participants and their caregivers will attend multiple visits where cognitive assessments and interviews will be performed. Key measures include changes in a cognitive scale (ADAS-Cog11) and clinical impressions of improvement at 24 weeks. Caregivers play an important role by providing information, ensuring medication adherence, and participating in study activities. Safety and treatment effects will be carefully monitored throughout the trial.

Researchers are evaluating the effectiveness and safety of remibrutinib in adults aged 18 to 65 years with secondary progressive multiple sclerosis (SPMS). This Phase III study is randomized, double-blind, and placebo-controlled, designed to better understand how remibrutinib affects disability progression in SPMS patients over time. Participants will be randomly assigned to receive either oral remibrutinib tablets or matching placebo tablets during the Core Part of the study, which is event-driven and double-blinded. After this period, all participants may enter an Extension Part where they receive open-label remibrutinib treatment. This design allows researchers to compare remibrutinib against placebo and then monitor long-term effects when all participants receive the active drug. Throughout the study, participants will undergo regular assessments including MRI scans and clinical evaluations to track changes in disability using the Expanded Disability Status Scale (EDSS). The primary outcome measured is the time to confirmed disability progression over six months, with follow-up lasting up to approximately five years. Safety, tolerability, and other health parameters will also be closely monitored during both study phases.