Le Chesnay Rocquencourt

Researchers are studying acute pyelonephritis (AP), a common bacterial kidney infection in children, focusing on those aged 1 month to less than 3 years without prior urological malformations. The study compares a short 3-day intravenous (IV) antibiotic treatment alone to a 3-day IV treatment followed by 7 days of oral antibiotics. The goal is to see if the shorter IV-only treatment is as effective at preventing infection recurrence and long-term kidney scarring, while possibly reducing antibiotic resistance and preserving gut microbiota diversity. Participants receive either IV ceftriaxone and/or amikacin once daily for 3 days, or the same 3-day IV treatment followed by 7 days of oral cotrimoxazole or cefixime. The study includes procedures like procalcitonin testing and fecal or rectal swabs collected at several points during and after treatment (day 0, 3, 10 or 17, and 31 or 38) to monitor bacterial presence and gut microbiota changes. Treatment begins after initial confirmation of infection and favorable early response. During the study, children are closely monitored for infection recurrence 28 days after completing antibiotics. Assessments include clinical evaluations, urine cultures, and monitoring for any adverse effects. The total participation covers treatment and follow-up periods to ensure safety and measure outcomes such as infection recurrence and bacterial resistance. This is a Phase 4 open-label randomized trial conducted across multiple centers.

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 evaluating efruxifermin (EFX) in adults aged 18 to 80 who have compensated cirrhosis caused by nonalcoholic steatohepatitis (NASH) or metabolic dysfunction-associated steatohepatitis (MASH). This Phase 3, randomized, double-blind, placebo-controlled study aims to assess the safety and effectiveness of EFX in improving liver health and delaying disease progression in this population. The study focuses on subjects with advanced liver fibrosis (stage 4) but without liver decompensation. Participants are randomly assigned to receive either efruxifermin or a placebo, both administered by subcutaneous injection. The study includes two cohorts: Cohort 1 requires biopsy confirmation of liver fibrosis and specific metabolic features, while Cohort 2 allows biopsy or non-invasive diagnosis. Treatment and observation continue over an extended period to evaluate changes in liver fibrosis and clinical events. During the study, researchers will monitor the time until significant clinical events such as disease progression or liver decompensation occur, with a follow-up of up to five years. For Cohort 1, the proportion of participants showing improvement in fibrosis without worsening steatohepatitis will be assessed at 96 weeks. Participants will undergo regular evaluations including clinical assessments and laboratory tests to track liver function and safety throughout the study period.

Researchers are conducting a global Phase 3 study to compare the effectiveness and safety of olverembatinib combined with chemotherapy versus the investigator's choice of tyrosine kinase inhibitor (TKI) combined with chemotherapy in patients newly diagnosed with Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia (Ph+ ALL). This study aims to evaluate these treatments in a randomized and open-label setting to determine the best approach for this condition. Participants will receive treatment in one of two groups: one group will take olverembatinib orally every other day along with chemotherapy, while the other group will receive an investigator-chosen TKI orally once daily combined with chemotherapy. Both treatments are studied over cycles of 28 days, focusing on the initial three cycles for measuring response. During the study, participants will be monitored for treatment effects, including the rate of minimal residual disease negative complete remission from cycles 1 to 3. Researchers will assess safety, treatment adherence, and any side effects. The study includes various evaluations to ensure participant health and treatment effectiveness over the course of therapy.

Researchers are evaluating the safety, effectiveness, and tolerability of subcutaneous blinatumomab for treating Relapsed or Refractory B cell Precursor Acute Lymphoblastic Leukemia (R/R B-ALL) and Minimal Residual Disease Positive (MRD+) B-ALL in participants aged 12 years and older. This Phase 1/2 study aims to find the maximum tolerated dose and recommended dose for Phase 2, as well as to assess clinical pharmacokinetics of two blinatumomab formulations (SC1 and SC2). Blinatumomab will be given as a subcutaneous injection. The study includes several parts: a dose escalation phase to determine safety and dosage, followed by dose expansion and Phase 2 cohorts to further evaluate efficacy and drug concentrations. Different participant groups will receive treatments based on their disease status, with monitoring periods ranging from up to 29 days for early toxicities to approximately 28 weeks for adverse events and up to 10 weeks for remission outcomes. Participants will undergo regular assessments including safety evaluations, response to treatment, and pharmacokinetic measurements. Researchers will monitor for treatment-related side effects, remission status, and drug levels in the blood. The study involves ongoing follow-up to capture complete remission rates, disease response, and safety for up to several months after treatment begins.

Researchers are evaluating the safety and effectiveness of brenipatide at different doses compared with a placebo in adults with uncontrolled moderate to severe asthma. This Phase 2 study focuses on participants who have a history of asthma requiring controller medication and recent severe asthma exacerbations. The goal is to better understand how brenipatide impacts asthma control over an extended period. Participants will receive either brenipatide or a placebo, both administered by subcutaneous injection. The study includes a 52-week treatment period during which the effects of the drug on asthma exacerbations and symptoms will be monitored. This randomized, double-blind approach helps compare the responses between the treatment and placebo groups. Study involvement lasts about 65 weeks, covering screening, treatment, and follow-up phases. During the study, researchers will assess participants' asthma control using questionnaires and track the annual rate of asthma exacerbations. Safety and treatment responses will be closely monitored throughout the trial to evaluate the drug's impact and participant well-being.

Researchers are conducting a Phase 3, multicenter, open-label study to evaluate the combination of tobevibart and elebsiran in adults with chronic Hepatitis D Virus (HDV) infection who have not achieved viral suppression with bulevirtide. The study focuses on participants aged 18 to 70 years who have ongoing HDV infection despite prior treatment with bulevirtide and aims to assess the safety and effectiveness of the new combination therapy. Participants receive tobevibart and elebsiran, both given by subcutaneous injection. All participants have been on bulevirtide injections for at least 24 weeks before joining the study. The study monitors viral load to determine if HDV RNA becomes undetectable at Week 24 and again 24 weeks after treatment ends. This design allows researchers to evaluate how well the combination therapy suppresses the virus over time. During the study, participants undergo regular assessments including HDV RNA measurements to track viral levels. The main outcomes focus on whether the virus is below the lower limit of detection at specific time points during and after treatment. Safety and tolerability are also monitored throughout the trial. Total participation spans the treatment phase and follow-up periods needed to observe sustained viral suppression.

Researchers are evaluating the effectiveness of brelovitug (BJT-778) compared to bulevirtide for treating chronic hepatitis delta infection (CHD). This Phase 3, global, randomized, open-label, multicenter trial aims to assess long-term treatment options for patients with chronic HDV infection. The study focuses on measuring virologic response and normalization of ALT levels as key outcomes. Participants will be assigned randomly in a 3:1 ratio to one of two treatment groups. One group will receive brelovitug 300 mg by subcutaneous injection once weekly for 96 weeks. The other group will receive bulevirtide 2 mg by subcutaneous injection daily for 48 weeks, followed by brelovitug 300 mg once weekly for the next 48 weeks. These two arms allow comparison of brelovitug alone versus a sequential bulevirtide and brelovitug treatment. Throughout the study, participants will be monitored for virologic response and ALT normalization at Week 48 as the primary outcome. The study includes regular blood tests and clinical evaluations to assess liver function and viral levels. Safety and treatment adherence will also be followed during the nearly two-year participation period to evaluate the long-term effects of these therapies.

Researchers are evaluating the adaptation of the FACETS fatigue management program, which combines cognitive-behavioral therapy and energy conservation strategies, specifically for adults with sickle cell disease experiencing fatigue. This adapted program is called the Drépa-FACETS program and targets adult patients with major sickle cell syndrome regardless of genotype. Participants will engage in the Drépa-FACETS program designed for sickle cell disease. The program includes sessions and home exercises to help manage fatigue. While the exact schedule is not detailed, the study focuses on implementing and assessing the feasibility of this program. Participants' involvement includes giving oral consent and having sufficient French language skills to complete assessments, questionnaires, and follow the program. Researchers will measure the feasibility of the program over approximately 10 weeks, monitoring participants from enrollment through the end of the protocol visits. Safety and eligibility are carefully monitored, excluding those with severe psychiatric conditions, other fatigue-causing chronic diseases, vaso-occlusive crises, hospitalizations, or legal protection status.

Researchers are studying the biological features of advanced ALK-rearranged non-small cell lung cancer (NSCLC) in patients treated with new generation tyrosine kinase inhibitors (TKIs) as their first therapy. This study is part of the national EXPLORE ALK cohort, a multi-center observational project in France, focusing on patients with this specific genetic alteration. The goal is to better understand the tumor biology and resistance mechanisms by analyzing samples from diagnosis through disease progression. The study collects tumor tissue samples at diagnosis and, when possible, at disease progression for RNA sequencing to identify ALK fusion partners, variants, and co-mutations. Blood samples are also taken at diagnosis, first tumor evaluation, and at progression to analyze circulating tumor DNA (ctDNA) using next-generation sequencing panels that detect mutations, fusions, and other genetic changes. These biological analyses are centralized at specialized centers such as the Léon Bérard Center and Rouen University Hospital. Patients are treated with approved ALK inhibitors like alectinib, brigatinib, lorlatinib, or entrectinib as part of their standard care. Participants will provide blood samples at multiple time points and, if possible, tumor biopsy samples for detailed genetic analysis. Researchers will monitor the progression-free survival from treatment start for up to 72 months. The study involves regular evaluations to assess tumor status and collect biological material to track genetic changes over time. Consent for sample collection and participation in the study is required, and patient data is managed within the national health system framework.