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 efruxifermin EFX in a randomized, double-blind, placebo-controlled Phase 3 study involving adults aged 18 to 80 with compensated cirrhosis caused by Nonalcoholic Steatohepatitis NASH or Metabolic Dysfunction-Associated Steatohepatitis MASH. The study aims to assess the safety and efficacy of EFX in preventing disease progression and liver complications over a long-term period. Participants receive either efruxifermin or a placebo by subcutaneous injection. The study includes multiple groups, with one cohort requiring biopsy-proven compensated cirrhosis and specific metabolic features, and another allowing biopsy or non-invasive diagnosis. The treatment and follow-up last up to five years, with detailed monitoring at 96 weeks and beyond. During the trial, participants will undergo regular assessments of liver fibrosis, markers of liver injury, lipoproteins, insulin sensitivity, glycemic control, and body weight. Researchers will also monitor adverse events, laboratory tests, ECGs, ultrasounds, and vital signs throughout the study. The primary outcome measures include time to significant clinical events and improvements in fibrosis and steatohepatitis, with extensive safety and efficacy evaluations conducted over five 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 a placebo in adults who have uncontrolled moderate to severe asthma. The study focuses on how brenipatide may affect the frequency of asthma flare-ups over a one-year treatment period. This Phase 2, double-blind study is sponsored by Eli Lilly and Company and involves random assignment to treatment groups. Participants will 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 will receive the assigned injections regularly. The study includes a total participation time of about 65 weeks, encompassing screening, treatment, and follow-up phases. Throughout the study, participants will undergo various assessments including lung function tests, health questionnaires, and monitoring of asthma symptoms and medication use. Researchers will also track the presence of antibodies against the study drug and measure drug levels in the blood. Safety and treatment effects will be closely monitored from baseline through the study duration, with final evaluations occurring at week 52.

Researchers are evaluating the combination of tobevibart and elebsiran in adults aged 18 to 70 years with chronic Hepatitis D Virus HDV infection who have not achieved viral suppression with bulevirtide treatment. This Phase 3, randomized, open-label trial aims to assess the efficacy and safety of this combination therapy for participants who continue to have active HDV infection despite prior treatment with bulevirtide. The study is sponsored by Vir Biotechnology, Inc. and addresses a critical need for improved therapeutic options in this population. Participants will be assigned to one of two groups. One group will receive tobevibart plus elebsiran together for up to 240 weeks. The second group will continue bulevirtide treatment for 24 weeks before switching to the combination of tobevibart and elebsiran for the remaining 216 weeks. All drugs are given by subcutaneous injection. This design allows comparison of continued bulevirtide versus switching to the new combination therapy over a long-term period. During the study, participants will have their HDV RNA levels measured at multiple time points, including week 24, and up to 24 weeks after treatment ends, to evaluate viral suppression. Researchers will also monitor liver enzyme changes and record any treatment-related side effects through week 240. Safety assessments and laboratory tests will be conducted regularly throughout the study. The total duration for individual participation may be up to approximately 4.5 years, including extended follow-up to assess long-term outcomes and safety.

Researchers are evaluating two treatments, brelovitug BJT-778 and bulevirtide, for chronic hepatitis delta infection CHD in a global Phase 3 study. The main purpose is to compare the effectiveness of brelovitug to bulevirtide as long-term treatments for patients with chronic HDV infection. This open-label, randomized trial involves multiple centers worldwide to assess these therapies impact on viral response and liver health. The study has two treatment groups. One group receives brelovitug 300 mg by subcutaneous injection once weekly for 96 weeks. The other group receives bulevirtide 2 mg by subcutaneous injection once daily for 48 weeks, followed by brelovitug 300 mg once weekly for the next 48 weeks. Participants are randomly assigned in a 31 ratio to these arms to evaluate and compare the treatments over nearly two years. Participants will undergo regular assessments including blood tests to measure viral levels and liver enzymes. Researchers will monitor safety by recording adverse events up to 96 weeks and follow participants after treatment ends. Liver stiffness, quality of life, and other liver function scores will also be evaluated. The main outcome is the percentage of participants achieving virologic response and normalized liver enzymes at 48 weeks. The study aims to provide long-term data on these treatments effects on chronic HDV infection.

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.