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Researchers are collecting detailed clinical and biological information from patients starting anticancer immunotherapy to better understand immune-related side effects that can affect various organs. The study aims to identify factors that increase the risk of these toxicities and to develop ways to prevent or manage them effectively. This observational study will help personalize future immunotherapy treatments by balancing benefits and risks for each patient. Participants receive immunotherapy drugs such as checkpoint inhibitors including PD-1, PD-L1, CTLA4, and others either alone or with other cancer treatments. Patients are enrolled before their first immunotherapy treatment and followed for up to 5 years or until they stop treatment permanently. The study also collects blood, plasma, serum, and immune cells samples before, during, and at the onset of immune-related side effects. During the study, participants will be regularly monitored through clinical evaluations and biological sample collections to track any immune-related toxicities. Researchers will estimate the incidence of these side effects over 10 years. Data collected will include clinical details and biological markers to support further research on mechanisms causing toxicity. This long-term follow-up helps improve understanding of immunotherapy safety and guides future patient care.

Researchers are evaluating the safety, effectiveness, and how the body processes and responds to NXT007 prophylaxis compared with emicizumab prophylaxis in people aged 12 years and older who have severe or moderate congenital hemophilia A without factor VIII FVIII inhibitors, or any severity of hemophilia A with FVIII inhibitors. This phase 3, randomized, open-label study aims to compare these treatments to better understand their impact on bleeding rates and treatment burden. Participants will be randomly assigned to one of two main treatment groups. One group will receive NXT007 prophylaxis administered subcutaneously using an integrated drug-device combination product. The other group will receive emicizumab prophylaxis via subcutaneous injections, starting with weekly loading doses for 4 weeks, then maintenance dosing at various intervals depending on prior treatment status. After the main treatment period, participants from both arms can continue or switch to NXT007 in an open-label extension phase. Throughout the study, participants will be closely monitored with regular assessments, including measuring annualized bleed rates for different types of bleeds, treatment burden questionnaires, and safety evaluations such as adverse event monitoring and laboratory tests. These evaluations will continue throughout approximately 3.5 years of study participation to provide comprehensive data on treatment effects and safety.

Researchers are evaluating the efficacy and safety of trontinemab in people with early symptomatic Alzheimers disease, ranging from mild cognitive impairment to mild dementia due to Alzheimers. This Phase III trial aims to understand how trontinemab affects cognitive decline and disease progression in this population. Participants are randomly assigned to receive either intravenous trontinemab or a placebo in a parallel-group design. Treatment is administered by IV infusion, and the effects are compared over a period of 72 weeks. The study includes comprehensive safety and efficacy assessments throughout this period. During the 72 weeks of the study, participants will undergo various evaluations including cognitive tests such as the Clinical Dementia Rating-Sum of Boxes CDR-SB, Alzheimers Disease Assessment Scales, brain imaging with PET and MRI scans, and biomarker measurements in cerebrospinal fluid and blood. Safety monitoring includes tracking adverse events, infusion reactions, and antibody development. The study requires participants to have a study partner and to complete all study procedures over this time.

Researchers are studying people with idiopathic pulmonary fibrosis IPF or progressive pulmonary fibrosis PPF who have previously taken nerandomilast in another study. The aim is to assess how well patients tolerate long-term treatment with nerandomilast and to evaluate whether it improves lung function and delays worsening symptoms, hospital visits, or death. This open-label extension trial is sponsored by Boehringer Ingelheim and focuses on treatment over an extended period. Participants take nerandomilast tablets for up to 1 year and 10 months while continuing their usual pulmonary fibrosis treatments. The study involves a single treatment group receiving the drug, and no placebo or comparison groups. Regular visits with doctors help monitor health and collect data during this extended treatment phase. Throughout the study, participants undergo regular lung function tests and health assessments to track any adverse events and changes in lung capacity. The main outcome measured is the occurrence of any adverse events for up to about 99 weeks. Secondary outcomes include changes in forced vital capacity and time to worsening of disease symptoms or hospitalization. The study includes ongoing safety monitoring with a total participation time of up to nearly two years.

Researchers are studying the long-term safety and effects of ritlecitinib, an oral medicine, for children with severe alopecia areata, a condition that causes significant hair loss. This Phase 3 trial focuses on participants who completed previous Pfizer studies and have notable scalp hair loss or specific vaccination history. The study aims to understand how well ritlecitinib is tolerated and its impact on hair regrowth and quality of life over time. Participants will take ritlecitinib capsules once daily at home for up to three years. Those who previously received different doses of ritlecitinib will continue the same dose, while others will be randomly assigned to either a higher or lower dose. Dose adjustments and treatment continuation will be evaluated at specific months, with some participants switched to placebo if they do not meet efficacy criteria, though they remain in the study. During the trial, participants will attend 17 clinic visits and receive monthly phone calls. Researchers will monitor safety by tracking adverse events and assess effectiveness using tools like the Severity of Alopecia Tool SALT, eyebrow and eyelash assessments, and patient-reported outcomes on anxiety, depression, behavior, and quality of life. The study includes ongoing safety checks and measures cognitive function at select points, ensuring thorough evaluation throughout the three-year participation period.

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.

This research aims to better understand the natural history of Congenital Myasthenic Syndromes CMS caused by mutations in the DOK7, MUSK, AGRN, or LRP4 genes. It evaluates disease activity by collecting clinical data and quality of life information from participants with these specific genetic mutations. The study is observational and involves participants aged 2 years and older diagnosed with CMS due to these mutations. Participants will attend up to four study visits where clinical assessments will be performed. These assessments include evaluating symptoms and quality of life using tools like the Quantitative Myasthenia Gravis QMG score and other standardized measures. The study collects both retrospective and prospective data on diagnosis, healthcare use, medications, and health status changes related to CMS over a period of up to 12 months. During the study, participants will undergo various evaluations including symptom scoring, questionnaires on daily living activities, fatigue, and health-related quality of life. Researchers will analyze changes from baseline in these measures to understand disease progression and impact. No investigational treatments are given, and the focus is on monitoring and documenting the condition. Participation may last up to 12 months with scheduled visits for data collection and assessments.

Researchers are evaluating a new generation of magnetoencephalography MEG devices that use optically pumped magnetometers with Helium 4 to record brain magnetic activities. The study includes healthy adults and male athletes who have suffered a mild concussion. The goal is to compare this new devices ability to detect and map brain activity with that of classical MEG systems, focusing on brain responses to visual, auditory, somatosensory, and motor stimuli. Participants will undergo several experiments using the FYNA Research MEG system. Healthy volunteers will complete tasks involving visual and auditory attention, language production, rest, and visuo-motor activities. Concussed athletes will have their resting brain activity recorded to assess frequency modulations. The study compares signal-to-noise ratios and brain activity maps from the new device with those from classical MEG during these tasks. During the study, participants will visit once for inclusion and then return within 1 to 30 days for MEG recordings and assessments. Researchers will measure signal quality, brain activity mapping, and participant comfort through questionnaires after recording sessions. The study monitors safety and participant experience, aiming to gather detailed data on the new MEG devices performance. Total participation time depends on scheduling within the defined visit window.

Dravet Syndrome DS is a severe neurodevelopmental disorder caused mostly by mutations in the SCN1A gene, leading to frequent, drug-resistant seizures, cognitive deficits, and a high risk of sudden unexpected death in epilepsy SUDEP. SUDEP is a major cause of premature death in epilepsy patients and is linked to seizure frequency and brainstem dysfunction. This study aims to investigate serotonin 5HT pathway alterations in the brainstem of adults with DS using PET and MRI imaging to better understand SUDEP mechanisms. The study will include 20 adult participants 10 with confirmed Dravet Syndrome, 10 with drug-resistant focal epilepsy, and 10 healthy adults. All participants will undergo PET-MRI imaging with injection of the tracer 18F-MPPF to assess the serotonin brainstem pathway. The imaging involves anatomical MRI scans followed by a 90-minute dynamic emission scan after tracer injection. Women of childbearing potential will have a pregnancy test before imaging. Participants will have one inclusion visit for eligibility review and clinical examination, followed by the PET-MRI scan scheduled within 2 to 8 weeks. Researchers will compare brainstem serotonin receptor binding between groups and relate imaging findings to central sleep apnea duration and brainstem volume. The study will measure the signal-to-noise ratio of 5-HT1A receptor binding in healthy controls and monitor safety throughout. Each participants involvement lasts from 2 to 8 weeks.

Researchers are evaluating CRD-4730, an oral drug, in a Phase 2 clinical trial involving adults with Catecholaminergic Polymorphic Ventricular Tachycardia CPVT. This study aims to assess the safety, tolerability, pharmacokinetics PK, and pharmacodynamics PD of CRD-4730. Participants will be part of a randomized, double-blind, placebo-controlled crossover study to better understand how CRD-4730 affects CPVT symptoms and treatment responses. Participants will be randomly assigned to one of three sequences in a 3-period crossover design. Each participant will receive two different doses of CRD-4730 and one matching placebo dose in a random order. The study includes multiple dosing periods where participants take tablets of CRD-4730 or placebo, allowing researchers to compare the effects and tolerability of different doses against placebo. During the trial, participants will undergo various assessments to monitor safety, drug levels, and effects on their condition from baseline to Day 101. The study involves close monitoring of heart function and other health measures, with outcome evaluations occurring at multiple timepoints. This will help researchers understand how the drug behaves in the body and its impact on CPVT symptoms, with the trial lasting several months to complete all study periods and follow-ups.