Pontault-combault, France

Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients with Homozygous Familial Hypercholesterolemia

This is a multicenter, international, long-term observational study investigating the real-world impact of lomitapide on Major Adverse Cardiovascular Events (MACE) in patients with Homozygous Familial Hypercholesterolemia (HoFH). Study Design: Observational, open-label, retrospective and prospective study Data will be collected from >26 lipid centers across Europe Patients will serve as their own control, with comparisons between pre-treatment (3 years before lomitapide) and post-treatment (first 3 years of lomitapide therapy) periods Study Population: Approximately 72 adult patients (≥18 years) diagnosed with HoFH Patients must have received lomitapide for at least 12 months Availability of 3 years of pre-treatment clinical records Objectives: Primary Objective: Evaluate the incidence of MACE before and after lomitapide treatment Secondary Objectives: Assess changes in LDL-C, total cholesterol, liver function tests (ALT, AST, GGT), and lipid-lowering therapy usage (e.g., discontinuation of LDL apheresis, addition of PCSK9 inhibitors) Endpoints: Primary Endpoint: Change in MACE incidence over the 3-year treatment period Secondary Endpoints: Changes in lipid levels, liver safety markers, and adherence to treatment protocols Safety Considerations: The study follows real-world clinical practice, with monitoring of adverse events, including liver-related safety concerns associated with lomitapide Data will be collected in an electronic Case Report Form (eCRF) and analyzed following Good Clinical Practice (GCP) guidelines This study aims to generate real-world evidence on the cardiovascular impact of lomitapide in HoFH patients, addressing an unmet clinical need for data on long-term outcomes.

Evaluation of the "Intranasal Fentanyl" Protocol in Pediatric Surgical Emergencies at Strasbourg University Hospital

A Study to Learn About How Safe BAY 3389934 is, Its Suitable Dose, and How it Affects the Participants With Sepsis Induced Coagulopathy

Long-Term Evaluation of TAILORED Vs Anatomical Ablation Strategy for Persistent Atrial Fibrillation

The TAILORED-AF study demonstrated at one year's follow-up that a tailored ablation strategy guided by Volta Medical AI-software targeting areas of spatio-temporal dispersed electrograms in combination with pulmonary vein isolation (PVI) ablation is more effective to an anatomical ablation strategy targeting PVI alone (current standard of care) for the treatment of persistent atrial fibrillation (AF). VX1 legacy device renamed Volta AF-Xplorer was used in the TAILORED-AF study in the treatment arm. No additional treatments are specifically required in the scope of this ancillary TAILORED-LT study which aims to follow the patients previously treated in the initial TAILORED-AF study over the long-term. However, in the event of repeat procedures during the TAILORED-LT study, the choice of the ablation technique will be left to the investigator's discretion, regardless of the patient's randomization arm in the TAILORED-AF study. The annual follow-up will be performed as in routine clinical practice post AF ablation procedures: visits at 24 months, 36 months, 48 months and 60 months post TAILORED-AF Study index procedure. It is possible that some of these annual visits cannot be completed prospectively due to time already having elapsed between the end of the subject participation in the TAILORED-AF study and the date of enrollment in the extension TAILORED-LT study. In this case, available data (among those expected by the study protocol) will be collected retrospectively. The additional procedures related to this clinical investigation are limited to annual 24-hour Holters and to the administration of Quality Of Life questionnaires (SF-36 and AFEQT) to the patients during follow-up visits.

The Objective of This Study is to Evaluate the Perfomance of a Photorefraction Device for Screening Ametropia in Children. Eligible Participants Will Undergo Multiple Refractive Error Measurements, Visual Acuity and Strabismus Assessments During a Single Evaluation Visit.

Participants after verification of their eligibility and informed consent, participate to a single evaluation visit during which several objective refraction measurements are performed with the investigational device, a tabletop refractometer and a comparable vision screener with and without cyclopegia. Additionally far vision visual acuity, strabismus assessments and subjective refraction measurement are also performed by investigators.

A Clinical Study of Neflamapimod in Patients with Dementia with Lewy Bodies

Intradialytic Exercise and Remote Ischaemic Preconditioning: a Cardioprotective Role ?

Antimicrobial Therapy for Difficult-to-treat Pseudomonas Aeruginosa

Infections due to Pseudomonas aeruginosa isolates with acquired resistances to all first-line antipseudomonal beta-lactams and fluoroquinolones (difficult-to-treat isolates - DTR), pose serious therapeutical challenges, especially in critically ill and/or immunocompromised patients. Certain new beta-lactam/beta-lactamase inhibitor combinations (BL/BLI (beta lactamine/ beta lactamase inhibitor) - i.e., ceftolozane-tazobactam, ceftazidime-avibactam, imipenem-relebactam, others) and cefiderocol have shown promising results for the treatment of infections due to DTR P. aeruginosa. However, multicenter data on their real-life utilization in this indication are still scarce. The ADDICT study is a prospective, multicenter cohort study including unselected patients with DTR P. aeruginosa infection requiring definite intravenous antimicrobial therapy. The primary objective of the study is to investigate the clinical efficacy of available options (new BL/BLI, cefiderocol or older agents such as aminoglycosides and colistin) in this population. Secondary objectives are to compare the clinical and microbiological efficacy of available options in infections due to DTR P. aeruginosa with in vitro susceptibility to more than one last-resort drug, to compare the incidence of non-ecological adverse events observed with these drugs, to assess the incidence of resistance emergence under therapy and to elucidate the molecular mechanisms of resistance emergence, to assess the benefits and risks of combination therapy in this indication, to compare the acquisition rates of multidrug-resistant bacteria other than DTR P. aeruginosa, and Clostridioides difficile infection, to compare Day-28 and in-hospital all-cause mortality rates. Patients will be recruited in 60 hospital centers contributing to four French networks of research in infectious diseases and critical care (CRICS-TRIGGERSEP, ReaRezo, OutcomeRéa, RENARCI - PROMISE metanetwork). Clinical variables will be collected through an electronic case-report form. DTR P. aeruginosa isolates will be sent to the National Reference Center of Antimicrobial Resistance in P. aeruginosa for centralized analyses (extended antimicrobial susceptibility testing, MLST, whole-genome sequencing of successive isolates if resistance emergence under therapy).

First-in-human Integrated Endoscopic Ultrasound (EUS) Navigation System Clinical Study

Since its inception in the 1980s, the field of endoscopic ultrasound (EUS) has experienced significant expansion and evolution and is now a pivotal component of pancreatobiliary assessment. Proficiency in EUS necessitates a synthesis of cognitive and technical competencies encompassing endoscopic manipulation, image interpretation, and an extensive comprehension of intraabdominal anatomical structures. Acquisition of these competencies is a progressive and highly operator-dependent process, and thereby necessitates an extended learning curve for practitioners. The American Society for Gastrointestinal Endoscopy (ASGE) has recommended the performance of a minimum of 190 supervised EUS for practicing autonomous EUS. However, the compliance of these parameters does not guarantee competence achievement, and there is a wide variability between endoscopists leading to disparities in the techniques' quality and irregular outcomes. The "EZ-EUS" system is designed to help the operator understand the orientation and the position of the probe tip in the patient. This system offers navigation similar to that of the Global Positioning System (GPS), but for EUS procedures. To achieve this, it uses a 3D model based on scanner imaging data recorded before the procedure. In this study, the intended purpose of the "EZ-EUS" system is to assist echo-endoscopy operators in accurately locating the pancreatic gland, conducting a comprehensive evaluation of the region of interest, and facilitating the detection and identification of lesions within it. We hypothesize that this tool will streamline procedure times and enable comprehensive imaging of the pancreas and its lesions, thereby reducing the need for expert guidance for trainees and shortening the EUS learning curve. This study is exploratory, prospective, monocentric and will compare two randomized groups (with and without the EZ-EUS navigation system).

Description of a Retrospective Cohort of Patients With Transthyretin Cardiac Amyloidosis (ATTR-CA) in Internal Medicine