Garches

Researchers are evaluating the effects of iptacopan compared with a placebo in adults aged 18 to 85 years who have generalized Myasthenia Gravis positive for acetylcholine receptor antibodies (AChR+ gMG). This Phase III, randomized, double-blind, placebo-controlled, multicenter study aims to assess the efficacy, safety, and tolerability of iptacopan while participants continue their stable standard of care treatments. The study includes participants with moderate to severe gMG symptoms and positive diagnostic criteria. Participants will be randomly assigned in a 1:1 ratio to receive either iptacopan or a matching placebo in the form of hard gelatin capsules for six months (180 days). During this time, they will continue their stable standard of care treatments. After the double-blind treatment period, a maximum 60-month open-label extension phase is offered. Safety follow-up assessments will occur one week and one month after the last dose of study treatment. During the study, participants will be evaluated for changes in their Myasthenia Gravis Activity of Daily Living (MG-ADL) total score from baseline to month 6. Researchers will monitor safety and tolerability throughout the treatment and extension periods. Vaccination status, infection monitoring, and regular clinical assessments will be part of participant evaluations to ensure safety and track disease symptoms over time.

Researchers are evaluating the safety and effectiveness of increasing doses of IPN10200 to understand its pharmacodynamics and identify the best dose for treating adults with upper limb spasticity. This integrated Phase I/II, multicenter, double-blind, randomized study also compares IPN10200 with Dysport and placebo to find the optimal balance of efficacy and safety in adults aged 18 to 70 years with spastic hemiparesis following stroke or traumatic brain injury. Participants receive either IPN10200, Dysport, or placebo as a powder and solvent solution for injection. The study includes dose escalation and dose-finding phases to assess different dosing levels. Treatments are administered in the affected upper limb muscles, with eligibility based on specific muscle tone and spasticity angle criteria. The study monitors participants for up to 9 months, including a safety follow-up period. During the study, participants undergo regular assessments including vital signs (blood pressure and heart rate), clinical lab tests, physical examinations, and monitoring for treatment-emergent adverse events and antibodies to the study drugs. Researchers use these measures to evaluate safety and treatment effects over the 9-month period from baseline through the end of the study.

Researchers are evaluating how the body processes the drug nusinersen when delivered through a new implantable device called the ThecaFlex DRx System compared to the standard lumbar puncture (LP) method. This study focuses on participants with spinal muscular atrophy (SMA) and aims to understand the pharmacokinetic profile of nusinersen in these two delivery methods. The ThecaFlex DRx System is an investigational device consisting of a catheter connected to a port placed under the skin. Participants in this Phase 1 study will first receive a dose of nusinersen by lumbar puncture. Then, the ThecaFlex DRx System will be implanted, and participants will receive nusinersen through this device. The study includes a screening period of up to 30 days, which may overlap with the parent PIERRE study, and the total participation lasts about five months, overlapping with the first five months of the PIERRE study. Blood samples will be collected before and after each dose, including up to 24 hours post-dose, to measure drug levels. During the study, researchers will monitor the highest concentration of nusinersen in the blood and the amount of drug exposure over 24 hours after dosing. Participants will have blood drawn multiple times to assess these outcomes. The study will also track safety and treatment effects during this period to better understand how nusinersen behaves in the body when delivered by the ThecaFlex DRx System versus lumbar puncture.

Researchers are investigating a new treatment called BAY 3389934 for people who have sepsis-induced coagulopathy, a condition where an infection causes excessive blood clotting that can damage blood vessels and organs. This Phase 1 study aims to understand how safe BAY 3389934 is, determine the right dose, and observe its effects on patients being treated for sepsis-induced coagulopathy in an intensive care unit (ICU). Participants will receive BAY 3389934 as an intravenous infusion. They will be divided into two groups: the first group receives the lowest starting dose, and based on safety and tolerance, the dose may be adjusted. If no serious side effects occur, the second group will receive a higher dose. Each participant will be involved in the study for about 28 days. During the study, doctors will monitor participants closely by taking blood and urine samples, performing physical exams, checking vital signs like body temperature and heart rate, and examining heart health with electrocardiograms (ECG). Researchers will track any medical problems that arise during and after treatment, called adverse events, to evaluate safety. The main outcomes measured include the number and severity of treatment-emergent adverse events from the first dose up to 97 hours after stopping the treatment.

Researchers are evaluating the use of Mobile Stroke Units (MSUs) compared to standard care for patients with confirmed acute ischemic stroke in France. This academic-driven, open-label randomized controlled trial aims to assess both the medical and economic impact of deploying MSUs, with a particular focus on cost-effectiveness and patient outcomes measured three months after treatment. The study addresses concerns about the cost of MSUs and their effect on reducing time to mechanical thrombectomy, a key treatment for certain stroke patients. Participants will be randomly assigned to either receive care from a Mobile Stroke Unit, which is equipped with a CT scanner and can initiate intravenous thrombolysis before hospital arrival, or to usual care involving conventional ambulance services. The MSU allows prehospital brain imaging and more precise identification of patients who may need mechanical thrombectomy. The study plans to enroll 450 patients over three years, with follow-up at three months to evaluate outcomes and costs. During the study, patients will be monitored for clinical outcomes including functional status using the Modified Rankin Scale at three months. Costs and health benefits will be tracked to estimate the incremental cost-utility ratio over the participants' lifetimes. The trial involves blinded assessment of efficacy endpoints and collects data prospectively. Overall participation includes emergency call, treatment allocation, and systematic follow-up for outcome and economic assessments.

Researchers are evaluating the effectiveness of different antimicrobial treatments for infections caused by difficult-to-treat Pseudomonas aeruginosa bacteria. This infection is especially challenging for patients who are critically ill or have weakened immune systems. The study focuses on comparing new beta-lactam/beta-lactamase inhibitor combinations, cefiderocol, and older drugs like aminoglycosides and colistin in real-life clinical settings across multiple hospital centers in France. Participants will receive intravenous antimicrobial therapy tailored to treat their difficult-to-treat P. aeruginosa infection. The study observes the use of new and older antimicrobial drugs to assess their clinical efficacy. Patient data and bacterial samples will be collected and analyzed centrally to better understand drug resistance mechanisms and treatment outcomes. Participants will be monitored for clinical cure shortly after completing therapy and on Day 7 ± 2 days. Researchers will collect clinical information through electronic case-report forms and send bacterial isolates to a national center for detailed testing. Outcomes include cure rates, resistance development, adverse events, and mortality rates, with follow-up during hospitalization and up to 28 days after treatment. The study aims to provide valuable real-world data on treating these challenging infections.

Acute bronchiolitis is a common illness affecting children under two years old, mainly caused by the respiratory syncytial virus (RSV). It is often hard to predict how severe the infection will be when symptoms first appear. Some studies suggest that the body's microbiota, including the intestinal, oral, and nasal bacteria, as well as the immune response to RSV, play important roles in the disease course. This study involves 80 children under 12 months old who have bronchiolitis during the RSV epidemic season. Both hospitalized and non-hospitalized children will be included. Researchers will collect a single set of samples from the mouth, nose, and stool to study the microbiota and look for imbalances called dysbiosis. A small blood sample will also be taken to examine the immune response. Participants will be monitored through these samples to assess the presence of dysbiosis and immune factors related to bronchiolitis. The study focuses on understanding the relationship between microbiota changes and how the immune system reacts during bronchiolitis. This information may help improve future care and management for infants with this condition.

People with cognitive, neuromotor, or sensory impairments often face multiple barriers to physical activity, including personal, organizational, societal, and interpersonal challenges. This study aims to describe these barriers and the factors leading to reduced performance in competitive sports as patients age. The study focuses on understanding how these obstacles change over time and the impact of medical and social treatments, which is currently unknown. Researchers are conducting an ambispective, single-center cohort study involving patients with these impairments who are seen at the Parasport Health Unit of the Physical Medicine and Rehabilitation Department. Participants receive standard care including clinical, radiological, biological, and physiological examinations. Data collection includes medical records and clinical activities both inside and outside the hospital. Participants will be monitored through their medical records and clinical assessments, with data collected on their health status and examinations. The main outcome measured is the change in barriers to physical activity during aging over approximately one year. This study involves patients aged 6 years and older, with no upper age limit specified, and includes follow-up of their physical activity challenges and related health information.

Infantile spasms are seizures marked by rapid, repeated contractions of muscles, usually involving sudden body flexion or extension and sometimes eye movements. These spasms are brief but can occur in series lasting several minutes. Early identification and treatment are crucial since delays often lead to cognitive decline. Diagnosing infantile spasms typically requires video-EEG, which is expensive and not always accessible, especially in developing areas. This research focuses on developing an automated video analysis system using computer vision and learning models to detect spasms from simple smartphone or webcam videos, aiming to improve early diagnosis and monitoring. The project uses existing video-EEG recordings from pediatric neurophysiology labs in the Paris region to train the system to recognize spasms confirmed by specialists. The goal is to reach over 95% sensitivity and specificity to make this tool practical for healthcare professionals and parents, enabling widespread screening and faster referral for treatment. The study involves analyzing stored videos of children with spasms and other epileptic seizures to refine the system's accuracy. Participants are children who have undergone video-EEG in participating centers and have recorded spasms or other seizures. Researchers will evaluate the system's ability to detect spasms accurately compared to the gold-standard video-EEG diagnosis. The primary outcome is achieving automated detection of spasms with over 95% accuracy at two years. This retrospective study uses existing data to demonstrate proof-of-concept before future prospective studies. Results will be shared through scientific publications.

Researchers are studying how extracorporeal blood purification using the oXiris hemofiltration membrane affects patients with vasoplegic shock who need hemodynamic support. This pilot, non-interventional study explores how this treatment may improve blood flow and clear certain vasoactive metabolites called angiotensin peptides, which are involved in controlling blood vessel tone. The study builds on earlier findings suggesting that oXiris treatment can reduce the need for vasopressors in both children and adults with septic or vasoplegic shock. The treatment involves using the oXiris membrane with the Prismaflex system, along with dialysis solutions like Phoxylium or He9mosol B0, delivered through a dialysis catheter. Anticoagulation is managed with heparin, citrate, or none if bleeding risk is high. The treatment volume is up to 35 ml/kg/h. Blood samples are taken before treatment, at about 24 hours, and at 72 hours to measure angiotensin peptides and related enzymes using specialized lab techniques. Participants will be monitored for 3 months to track outcomes including survival. Data from medical records and lab tests are collected and analyzed to understand how oXiris therapy affects hemodynamic stability and vasoactive metabolite clearance. The main outcome measured is a 50% reduction in vasoinotropic score within 24 hours after treatment starts.