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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.

This research aims to evaluate the real-world effectiveness of deucravacitinib treatment in adults diagnosed with moderate-to-severe plaque psoriasis. The study is conducted in France and focuses on understanding how this treatment performs outside of controlled clinical trial settings. Participants in this observational study will be newly starting deucravacitinib as prescribed by their treating clinician. There are no additional study treatments or placebo groups, as the study observes the outcomes of the treatment during routine clinical care. During the study, researchers will assess clinical outcomes including the Physician's Global Assessment (PGA) and the Dermatology Life Quality Index (DLQI) at baseline and at months 4, 12, 18 (optional), and 24. They will also monitor how long participants remain on deucravacitinib treatment, up to 24 months. These evaluations help to measure both the effectiveness and impact on quality of life for participants with plaque psoriasis.

Researchers are evaluating the safety and effectiveness of bomedemstat (MK-3543) compared with the best available therapy (BAT) in adults with essential thrombocythemia (ET) who have not responded well to or cannot tolerate hydroxyurea. This phase 3 clinical trial aims to determine if bomedemstat provides a better durable clinicohematologic response in these participants. Participants will receive either bomedemstat as an oral capsule or one of the best available therapies, including anagrelide (oral capsule), busulfan (oral tablet), interferon alfa or its pegylated forms (subcutaneous solution), or ruxolitinib (oral tablet). The study involves a randomized, open-label design where treatments are compared directly. Throughout the study, participants will be monitored for their hematologic response up to about 52 weeks. Assessments include platelet and neutrophil counts before starting treatment to ensure eligibility. Safety and efficacy are tracked to evaluate the long-term impact of the treatments on ET.

Researchers are studying Philadelphia-negative myeloproliferative neoplasms (MPN), which include Polycythemia Vera (PV), essential thrombocythemia (ET), and prefibrotic myelofibrosis (PreMF). These chronic blood cancers involve specific mutations like JAK2V617F and carry a high risk of blood clots that can cause serious health problems. Current treatments include low-dose aspirin to reduce arterial clots, but patients still face risks of thrombosis and bleeding. This trial explores whether direct oral anticoagulants (DOACs), such as Apixaban or Rivaroxaban, might better prevent these clotting events in patients with the JAK2V617F mutation. Participants will be randomly assigned to receive either a DOAC (Apixaban 2.5 mg twice daily or Rivaroxaban 10 mg once daily) or low-dose aspirin (100 mg once daily). The study focuses on high-risk MPN patients with JAK2V617F mutation and will compare the effectiveness and safety of DOACs versus aspirin for preventing blood clots. Treatment will continue with close monitoring throughout the study. During the study, researchers will track the time until any arterial or venous blood clots occur over a 24-month follow-up period. Participants will undergo regular assessments to monitor clotting events, bleeding risks, and overall health. The trial aims to gather detailed information on how well these treatments prevent thrombosis and their safety profiles, helping to guide future care for patients with these blood disorders.

Chronic use of nitrous oxide can cause toxicity leading to neurological problems such as combined sclerosis of the spinal cord. This can result in difficulties walking or sensations like numbness and tingling, which may improve or worsen to the point of requiring a wheelchair. Recently, cases of blood clots have also been linked to nitrous oxide use. Blood or urine tests for nitrous oxide are not commonly done because the gas leaves the body quickly, so other markers like vitamin B12 and homocysteine levels are studied instead, though no official monitoring guidelines currently exist. The exact biological processes causing these health problems are still not well understood. The study involves collecting blood samples from participants for biological analysis to better understand markers related to nitrous oxide use. Participants include current or former recreational nitrous oxide users, both with and without clinical signs or biological effects from use. Blood samples are preserved for testing as part of routine care within the study framework. Participants will be monitored with blood tests to measure markers linked to nitrous oxide consumption over about one year. The main outcome measured is the change in blood markers related to nitrous oxide use during this period. The study requires participants to consent and be socially insured, and it excludes pregnant or breastfeeding women and those who have not used nitrous oxide recently or are unwilling to complete the study.

This research investigates acute myeloid leukemia (AML), a blood cancer marked by the rapid growth of abnormal myeloid cells. The trial focuses on patients newly diagnosed with de novo AML who have intermediate or adverse-risk genetic profiles. It explores the effectiveness of CPX-351 compared to standard intensive chemotherapy, particularly in patients with secondary-type AML mutations, which are linked to poorer outcomes with conventional treatments. Participants are randomly assigned to receive either CPX-351 or intensive chemotherapy. CPX-351 is given as induction therapy with doses on days 1, 3, and 5, followed by consolidation therapy on days 1 and 3. The intensive chemotherapy group receives cytarabine and idarubicin with specific dosing schedules for induction and consolidation phases. The study examines these treatments in phase II, aiming to assess remission rates and molecular responses. During the study, patients undergo bone marrow sampling to measure minimal residual disease using a specialized flow cytometry method after the first induction phase. Researchers monitor remission depth, treatment response, and safety throughout the treatment. Patients must be available for regular assessments, blood tests, and clinical management at the study center. The trial tracks outcomes like deep remission rates and explores biomarkers related to treatment response over the study period.

Researchers are evaluating whether using fungal biomarkers can help doctors stop antifungal treatment earlier in critically ill patients suspected of invasive Candida infections. The study aims to compare this biomarker-based approach to the usual care strategy, assessing if early discontinuation can safely reduce unnecessary antifungal use without increasing mortality by day 28. This is a randomized controlled open-label study involving patients who need empirical antifungal therapy for the first time in the ICU. Participants are divided into two groups. The intervention group will have their antifungal treatment duration guided by blood tests measuring (1,3)-Beta-D-glucan and mannan levels at the start of treatment and on day 3, with recommendations to stop treatment early if biomarker results allow. The control group will receive routine care based on international guidelines, typically involving 14 days of treatment if no proven infection occurs and the patient improves, or shorter durations in other cases. During the study, researchers will monitor when antifungal treatment is stopped, particularly noting if treatment ends before day 7 after it begins. They will also track patient outcomes up to day 28 to ensure safety. Participants must provide informed consent and are expected to stay in the ICU for at least 6 days after starting treatment. The main outcome measured is the percentage of patients who stop antifungal therapy early according to the study protocols.

Researchers are evaluating the effects of caffeine on cognitive decline in people with Alzheimer's disease at the beginning to moderate stages. This phase 3 trial aims to compare the impact of caffeine treatment versus placebo on cognition over 30 weeks. Alzheimer’s disease is a complex condition with no current cure, and caffeine's properties may offer symptomatic benefits, although high doses could cause anxiety and insomnia, especially in this vulnerable group. Participants will undergo a 6-week caffeine diet before starting treatment. Then, caffeine or placebo capsules will be given with a titration phase of 3 weeks increasing the dose by 100mg every stage until reaching a target of 400mg daily in two doses, maintained for 27 weeks. After treatment, doses will be gradually decreased following the same schedule. During the study, participants will be monitored for changes in cognitive function measured by neuropsychological tests at 30 weeks after randomization. Caregivers will be involved, and participants’ clinical status, safety, and adherence to a low caffeine diet will be assessed. The total participation duration includes the caffeine diet, titration, treatment, and dose reduction phases.

Researchers are conducting a multicenter, prospective, interventional clinical investigation in nine French healthcare establishments to assess the safety of the SECURIDRAP® SELFIA® bedding. This study follows the market withdrawal of the first version of this product and is carried out upon recommendation from the ASNM to evaluate the safety of the second version. The study involves patients with disorientation, cognitive impairment, or behavior disorders who have a medical prescription for the device. Participants will use the SECURIDRAP® SELFIA® bedding for 15 nights in real-life conditions within nursing homes and hospitals. During this time, an independent assessor will ensure that the device's usage conditions are properly followed. The study is interventional but non-comparative and is designed to pose minimal risks and constraints to participants. During the 15-night period, participants will be monitored for any adverse events related to the use of the SECURIDRAP® SELFIA®. Data collection will include safety evaluations to confirm the device's safe use in healthcare settings. The trial focuses on the occurrence rate of adverse events connected to the bedding's use, with close follow-up by researchers to ensure participant safety.

Chronic kidney disease (CKD) is a widespread health concern linked to high risks of mortality, cardiovascular disease, and progression to end-stage kidney disease, especially when paired with hypertension. Lowering blood pressure (BP) is known to reduce these risks, but optimal drug treatment strategies for patients with moderate to severe CKD and uncontrolled hypertension remain unclear. This trial evaluates whether a diuretic-based blood pressure lowering approach is more effective than usual care in reducing cardiovascular events, death, and kidney failure in this population. Participants will be assigned to either an antihypertensive treatment plan using a specific diuretic-based algorithm or to standard care for intensifying blood pressure control. All participants have moderate to severe CKD and uncontrolled hypertension despite treatment with at least one renin-angiotensin system blocker at the highest tolerated dose. The study compares these two approaches to blood pressure management over time, focusing on their impact on health outcomes. Throughout the study, participants will be monitored for up to 36 months to track the occurrence of end-stage kidney disease, significant declines in kidney function, cardiovascular events, and overall survival. Blood pressure control will be assessed by office, home, or ambulatory monitoring. Safety and treatment effects will be evaluated regularly to understand how these blood pressure strategies affect patients with CKD and uncontrolled hypertension.