Le Chesnay Rocquencourt

This research aims to evaluate antibiotic treatments for acute pyelonephritis AP in children aged 1 month to 3 years. It compares a shorter intravenous IV antibiotic course of 3 days alone to a longer treatment consisting of 3 days IV followed by 7 days of oral antibiotics. The goal is to see if the shorter IV treatment is as effective in curing AP and preventing recurrence and renal scarring, while reducing the risk of antibiotic resistance and preserving gut microbiota diversity. Participants are randomly assigned to one of two groups. The experimental group receives only 3 days of IV antibiotics with ceftriaxone andor amikacin, after which treatment is stopped. The control group receives the usual care of 3 days IV antibiotics followed by 7 days of oral antibiotics, either cotrimoxazole or cefixime. The study includes collection of fecal or rectal swabs and blood tests to assess microbiota and resistance. Treatment effectiveness and safety are compared between groups. During the study, children are monitored for fever, symptoms, and urine cultures to confirm infection clearance. Researchers measure recurrence of febrile urinary tract infections within 28 days after treatment ends and follow clinical cure at 10 or 17 days depending on the group. Additional assessments include monitoring recurrence at 90 days, antimicrobial resistance in gut bacteria, and intestinal microbiota diversity over about a month. Participation lasts through treatment and follow-up visits up to several weeks.

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.

Researchers are evaluating the safety and effects of a new medicine called NNC0487-0111 in people who have Heart Failure with preserved Ejection Fraction HFpEF or Heart Failure with mildly reduced Ejection Fraction HFmrEF and excess body weight. This phase 3 clinical trial aims to find out if NNC0487-0111 is safe and effective for treating these conditions compared to a placebo. Participants have HFpEF or HFmrEF and a body mass index of 30 or above. The study is sponsored by Novo Nordisk AS and uses a randomized, quadruple-masked design. Participants will receive either NNC0487-0111 or a matching placebo by injection under the skin once a week. The NNC0487-0111 is given in increasing doses over time. The study is parallel in design, meaning participants are randomly assigned to one of the two groups and receive that treatment throughout the trial. This treatment period extends for up to about 165 weeks. The study evaluates the time to certain heart failure events, hospitalizations, cardiovascular deaths, and other major cardiovascular events. During the study, participants will be monitored regularly to assess heart failure outcomes and kidney function, as well as quality of life using questionnaires like the Kansas City Cardiomyopathy Questionnaire. Safety and effectiveness are assessed through hospital visits, heart failure event tracking, and blood tests including kidney function and blood sugar levels. The total participation spans over three years, with ongoing evaluations to measure the time to heart failure events and cardiovascular outcomes. Participants receive close medical monitoring throughout the study period.

Researchers are evaluating efruxifermin EFX in a phase 3, randomized, double-blind, placebo-controlled study involving adults with compensated cirrhosis caused by NASH Nonalcoholic Steatohepatitis or MASH Metabolic Dysfunction-Associated Steatohepatitis. This study aims to assess the safety and effectiveness of EFX in preventing significant clinical events such as disease progression and liver decompensation over a period of up to 5 years. Participants are randomly assigned to receive either efruxifermin 50 mg or a placebo, both given by subcutaneous injection. The study includes two cohorts one with biopsy-proven compensated cirrhosis and specific metabolic scores, and another with biopsy-proven or non-invasive diagnosis of compensated cirrhosis. The study treatment and monitoring extend up to 5 years, with evaluations at 96 weeks and long-term follow-up to track liver fibrosis, markers of liver injury, insulin sensitivity, glycemic control, body weight, and safety outcomes. During the trial, participants undergo regular assessments including laboratory tests, ECGs, ultrasounds, and vital sign monitoring. Researchers will measure changes in liver fibrosis, steatohepatitis resolution, and metabolic markers throughout the study. Safety and tolerability are closely tracked by documenting adverse events and exposure duration. The study duration allows for long-term observation of treatment effects and disease progression, with participant involvement lasting up to 5 years.

Researchers are evaluating the safety and effectiveness of olverembatinib combined with chemotherapy compared to an investigators choice of tyrosine kinase inhibitor TKI combined with chemotherapy in adults newly diagnosed with Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia Ph ALL. This global, randomized, open-label Phase 3 study aims to provide important information about treatment options for this specific type of leukemia. Participants will receive either olverembatinib taken orally every other day along with chemotherapy, or a TKI chosen by the investigator taken orally once daily with chemotherapy. The study includes two groups one receiving the experimental olverembatinib combination and the other receiving the control treatment with investigator-selected TKI. Treatment cycles last 28 days, and the study follows participants through multiple cycles. During the study, participants will be monitored for their response to treatment by checking minimal residual disease negative complete remission rates during the first three cycles. Researchers will also assess event-free survival for up to 3 to 6 years, treatment-related side effects, drug levels in the blood, and quality of life related to leukemia. Safety assessments and other evaluations will continue through approximately two years of study participation, allowing for long-term monitoring of treatment effects and participant health.

Researchers are evaluating the safety and effectiveness of brenipatide at different dose levels compared with placebo in adults with uncontrolled moderate to severe asthma. This Phase 2 study aims to better understand how brenipatide may affect asthma symptoms and exacerbations over one year of treatment. Participants in this study are adults aged 18 to 75 years with a history of asthma and recent severe exacerbations. Participants will be randomly assigned to receive either one of two doses of brenipatide or a placebo, all administered by subcutaneous injection. The treatment period lasts 52 weeks, during which participants receive their assigned injections and are monitored regularly. The study includes a screening period before treatment and a follow-up period after treatment to assess ongoing safety and effects. During the study, participants will attend visits to complete questionnaires, lung function tests such as forced expiratory volume in one second FEV1, and assessments of asthma control and medication use. Researchers will monitor asthma exacerbation rates, rescue medication use, and the presence of anti-drug antibodies. The total study duration including screening, treatment, and follow-up is approximately 65 weeks.

This trial is a global, Phase 3, randomized, open-label study comparing brelovitug BJT-778 and bulevirtide for treating chronic hepatitis delta infection CHD. The main aim is to evaluate the long-term effectiveness of brelovitug versus bulevirtide in patients with chronic HDV infection. Approximately 172 participants will enroll to assess these treatments over an extended period. Participants are randomly assigned in a 31 ratio to one of two treatment arms. In the first arm, participants receive brelovitug 300 mg as a weekly subcutaneous injection for 96 weeks. In the second arm, participants receive bulevirtide 2 mg daily by subcutaneous injection for 48 weeks, followed by brelovitug 300 mg weekly for an additional 48 weeks. This design allows comparison of brelovitug alone to sequential treatment starting with bulevirtide. During the study, participants will have regular assessments including monitoring of viral load, liver enzymes, liver health markers, and quality of life questionnaires. Researchers will track treatment response, safety, adverse events, and liver disease progression over up to 96 weeks of treatment and follow-up. The main outcome measured is the percentage of participants achieving viral control and normalized liver enzyme levels at 48 weeks, with additional evaluations continuing through 96 weeks and post-treatment follow-up periods.

This research aims to adapt a fatigue management program called the FACETS program, which combines cognitive-behavioral therapy and energy conservation techniques, specifically for adult patients living with sickle cell disease. The study focuses on evaluating the feasibility of this adapted program, named Drpa-FACETS, for managing fatigue symptoms in this population. Participants will take part in the Drpa-FACETS program designed for sickle cell disease. The program includes sessions and home exercises tailored to help manage fatigue. The study will monitor participants from enrollment through the end of the protocol visits over a 10-week period. During the study, participants will complete assessments and questionnaires to measure fatigue and their ability to follow the program. The research team will evaluate how well the program works for adults with sickle cell disease by tracking adherence and outcomes related to fatigue management over the course of the study.

Researchers are studying advanced non-small cell lung cancer NSCLC with ALK gene rearrangement treated with next-generation tyrosine kinase inhibitors TKIs as first-line therapy. This prospective study, part of the national EXPLORE ALK cohort, aims to understand the biological characteristics and resistance mechanisms of this cancer type. It involves patients with stage IIIB or IV NSCLC who are not eligible for curative locoregional treatment and have confirmed ALK rearrangement. The study collects tumor tissue samples at diagnosis and at disease progression, if available, to analyze ALK fusion partners, variants, and co-mutations using RNA sequencing. Blood samples are taken at diagnosis, first tumor evaluation, and disease progression to analyze circulating tumor DNA ctDNA with next-generation sequencing. Treatments studied include alectinib, brigatinib, lorlatinib, and entrectinib, either marketed or under early access programs. Participants provide blood samples and allow use of tumor tissue for centralized biological analyses. Researchers measure progression-free survival up to 72 months as the primary outcome, along with overall survival, response rates, duration of response, ctDNA clearance, and resistance mechanisms associated with treatment and ALK fusion partners. The study involves regular evaluations over several years to monitor treatment outcomes and biological changes.

Researchers are conducting a national, multi-center observational study to understand the differences in autoimmune T and B lymphocytes among people with type 1 diabetes, other diabetes types or autoimmune conditions, and those without these diseases. The study aims to clarify how these immune cells contribute to type 1 diabetes development and to discover new biomarkers for diagnosis and treatment monitoring. This research is supported by the Institut National de la Sant Et de la Recherche Mdicale in France. Participants are grouped based on their diabetes status or autoimmune conditions, including those with type 1 diabetes, other types of diabetes, no diabetes, or those undergoing lymphadenectomy during abdominal surgery. Biological samples such as blood, stool, and lymph node tissues are collected to analyze immune cell characteristics. This study does not involve any treatment but focuses on detailed immune system profiling over six years. During the study, participants will provide biological samples and undergo evaluations to measure the frequency, phenotype, and activity of autoimmune lymphocytes. Researchers will compare immune cells in blood and pancreatic lymph nodes, identify novel autoimmune targets, and assess links between biomarkers and insulin secretion. The primary outcome is to define autoimmune T lymphocyte features related to islet antigens over six years, with secondary outcomes exploring B lymphocytes and pathogenicity. Participation involves ongoing monitoring and sample collection to better understand type 1 diabetes mechanisms.