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The number of patients with cancer receiving immunotherapy is growing, leading to new and unexpected side effects called immune-related adverse events (irAE) that can affect various organs. Managing these side effects requires a specialized, multidisciplinary approach and a professional network to provide timely and effective care. The study aims to create a comprehensive clinical and biological database called IMMUCARE-BASE to collect information on patients treated with anticancer immunotherapy, helping identify factors that increase the risk of toxicity and improve prevention and treatment strategies. Participants in this study will have their clinical data collected along with biological samples such as blood, plasma, serum, and peripheral blood mononuclear cells before treatment, during treatment, and if irAEs occur. The study includes patients starting immunotherapy alone or combined with other cancer treatments, with some eligibility for those who received immunotherapy previously more than six months ago. This database will support future personalized immunotherapy treatments based on individual benefits and risks. During the study, researchers will monitor the incidence of irAEs of all grades and organs over a period of 10 years. They will gather detailed clinical and biological data to understand mechanisms causing toxicity and to develop research programs focused on immunotherapy safety. The study involves ongoing follow-up and collection of samples to help improve care and anticipate adverse effects for patients receiving immunotherapy.

Researchers are evaluating the safety and effectiveness of trontinemab in people aged 50 to 90 with early symptoms of Alzheimer's disease, ranging from mild cognitive impairment to mild dementia. This Phase III clinical trial focuses on those who show evidence of Alzheimer's pathology and have a recent history of cognitive decline. The study aims to measure changes in cognitive function over 72 weeks. Participants will be randomly assigned to receive either intravenous trontinemab or a placebo. The trial is designed as a double-blind, placebo-controlled study, meaning neither participants nor researchers know who receives the active drug or placebo. The treatment period lasts up to 72 weeks, during which participants will undergo various assessments to monitor their cognitive status and safety. During the study, participants will complete clinical tests including cognitive assessments and imaging such as MRI, PET scans, or cerebrospinal fluid analysis to confirm Alzheimer's pathology. A study partner will assist participants as needed. Researchers will track changes from the start of the study through week 72 using tools like the Clinical Dementia Rating. Safety monitoring and adherence to study procedures will also be closely observed throughout the trial.

Researchers are evaluating the long-term safety and effects of nerandomilast in people with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF) who have previously completed treatment with nerandomilast in earlier studies. The study aims to understand how well participants tolerate nerandomilast over time, and whether it helps improve lung function, delays symptom worsening, reduces hospital visits, or impacts survival. This is a Phase 3 open-label extension trial. Participants take nerandomilast tablets daily for up to 1 year and 10 months while continuing their usual pulmonary fibrosis treatments. The study follows an open-label design where all participants receive nerandomilast. There are no placebo or comparator groups in this extension phase. Throughout the study, participants regularly visit their doctors for health assessments and lung function tests. Doctors monitor any health problems or side effects experienced during treatment. The main outcome measured is whether participants experience any adverse events up to the final follow-up visit, which occurs at week 99. This close monitoring helps evaluate the long-term safety and potential benefits of nerandomilast in this patient group.

Researchers are studying the long-term safety and effects of the medicine ritlecitinib for children with severe alopecia areata, a condition causing significant hair loss. This Phase 3 extension study involves participants who completed previous ritlecitinib studies and aims to assess long-term safety, effectiveness, and patient quality of life over up to 3 years. The study focuses on children aged 6 to 14 years who have experienced severe hair loss and have met specific prior study criteria. All participants receive ritlecitinib capsules taken once daily at home. Those who had higher or lower doses in a previous study continue the same dose, while those who previously received placebo or came from a different parent study are randomly assigned to a higher or lower dose. The study lasts up to 3 years and includes 17 clinic visits plus monthly phone calls to monitor treatment and health. During the study, participants are regularly evaluated for safety and treatment response using hair loss assessments and patient-reported measures of anxiety, depression, behavior, and skin-related quality of life. Safety monitoring tracks side effects and serious events from the start of consent through at least 28 days after the last dose. Participants are checked multiple times to decide whether to continue treatment, ensuring close observation throughout the trial.

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 participants with Congenital Myasthenic Syndromes (CMS) caused by mutations in the DOK7, MUSK, AGRN, or LRP4 genes. The goal is to understand symptoms, quality of life, and disease activity in these patients over time. This natural history study gathers important information about how CMS affects participants and tracks changes in their health. Participants will attend up to four study visits during which clinical assessments are performed. These assessments focus on evaluating symptoms and overall quality of life related to CMS. No specific treatments or interventions are administered, as the study is observational and aims to collect detailed data about the condition's progression. Throughout the study, researchers collect data retrospectively and prospectively over a period of up to 12 months. This includes information on diagnosis, healthcare use, medications, and changes in health status related to CMS. The study carefully monitors participants to better characterize the natural course of the disease and its impact on daily living.

Researchers are evaluating a new generation of non-invasive magnetoencephalography (MEG) devices that use optically pumped magnetometers with Helium 4 as the sensitive element (OPM He4) to record brain magnetic activity. The study involves healthy volunteers and athletes who have experienced a mild concussion. The main goal is to compare the new OPM MEG system's ability to detect brain signals with that of a classical MEG system, focusing on signal-to-noise ratio and the ability to reconstruct brain activity maps. The study includes four experiments using the FYNA Research system. Experiment 1 involves 20 healthy volunteers performing visual and auditory attention tasks to assess the system's recording of complex brain signal frequencies. Experiment 2 uses language production and resting tasks with healthy volunteers to evaluate functional brain mapping and resting network identification. Experiment 3 tests 20 healthy volunteers on a visuo-motor task to assess performance during movement, which affects spatial precision and low-frequency artifacts in the recordings. Experiment 4 involves 20 male athletes with mild concussion performing resting tasks to evaluate detection of brain activity modulations. Participants will be assessed during a visit scheduled 1 to 30 days after enrollment, where brain activity will be recorded using both classical MEG and the new FYNA Research system. Researchers will compare signal-to-noise ratios for visual, auditory, somesthetic, motor stimulations, and resting activity. The study includes informed consent, motivation assessment, and safety monitoring for contraindications to MEG and MRI. Total participation time and follow-up details are based on the intervention visit and evaluations during the study period.

Dravet Syndrome (DS) is a severe brain disorder caused mainly by mutations in the SCN1A gene, leading to difficult-to-control seizures, cognitive problems, and a high risk of sudden unexpected death in epilepsy (SUDEP). SUDEP often results from breathing problems after seizures and is linked to dysfunction in the brainstem serotonin system. This study aims to examine the serotonin brainstem pathway in adults with DS using PET and MRI imaging and compare findings with those in adults who have drug-resistant focal epilepsy and healthy controls to understand if these abnormalities are specific to DS or related to epilepsy in general. Participants will undergo a detailed clinical assessment followed by a PET-MRI scan within 2 to 8 weeks. The imaging includes anatomical MRI and dynamic PET imaging using the tracer [18F]-MPPF injected intravenously. The PET scan will last 90 minutes and capture brainstem serotonin receptor activity. Women of childbearing age will receive a pregnancy test before the imaging. The study will include 10 adults with DS, 10 with drug-resistant focal epilepsy, and 10 healthy adults. During the study, participants will be evaluated for eligibility, medical history, and clinical status. The primary outcome measures brainstem serotonin receptor binding over 90 minutes after tracer injection. The researchers will monitor safety, and participation will last between 2 to 8 weeks. The study aims to better understand brainstem serotonin changes in DS and epilepsy, potentially guiding future therapies.

Researchers are evaluating the safety, effectiveness, and how the body processes the drug etelcalcetide in children aged 2 to under 18 years who have secondary hyperparathyroidism (SHPT) and chronic kidney disease (CKD) while receiving hemodialysis. This Phase 3 study is designed to better understand how etelcalcetide works in this pediatric population, as it has been previously shown to help adult patients with similar conditions by controlling important blood chemicals. Participants will receive etelcalcetide during the study, which has been approved for adults with SHPT on hemodialysis. The study involves multiple doses with adjustments based on individual response. Patients must already be on hemodialysis or hemodiafiltration three or four times weekly for at least one month before starting. Treatments like vitamin D sterols, phosphate binders, and calcium supplements must be stable before and during the trial except for protocol-allowed adjustments. Throughout the study, participants will undergo blood tests to measure changes in parathyroid hormone levels from baseline between weeks 20 and 26. Other assessments include monitoring calcium and phosphorus levels, electrocardiograms, and safety evaluations. The study also tracks adherence to treatment and monitors for any side effects or health changes. The total duration and follow-up details are according to protocol schedules.

Researchers are evaluating the safety, tolerability, and effectiveness of LP352 in reducing seizures among children and adults diagnosed with Dravet Syndrome (DS). This phase 3, double-blind, randomized, placebo-controlled study aims to compare LP352 with a placebo to better understand its impact on seizure frequency in this population. The study involves participants aged 2 to 65 years and addresses the challenges patients with DS face due to various seizure types and treatment responses. Participants will receive either LP352 or a matching placebo, administered orally or through a feeding tube (G-tube or PEG tube). The study includes several phases: an initial screening period, followed by a titration period to adjust doses, then a maintenance period where treatment continues, and finally a taper period to gradually reduce treatment before a follow-up phase. The entire study duration is approximately 24 months. During the study, participants will be monitored for changes in the frequency of countable motor seizures compared to their baseline seizure activity over up to 15 weeks. They will be required to complete diaries throughout the study to track seizures and treatment adherence. Safety and tolerability will also be assessed throughout all study phases. The researchers will collect data on seizure counts and monitor participants' health to evaluate LP352's effects comprehensively.