Le Kremlin Bicetre

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 evaluating sotatercept as a potential treatment for pulmonary arterial hypertension (PAH), a condition where blood vessels in the lungs thicken and narrow, causing high blood pressure in the lungs and overworking the heart. PAH symptoms include difficulty breathing and reduced ability to be active. Current standard treatments address symptoms but do not stop disease progression. This Phase 3 study focuses on the long-term safety and tolerability of sotatercept when added to standard PAH therapy. Participants in this long-term follow-up study receive sotatercept through subcutaneous injections every three weeks. Only individuals who completed prior sotatercept PAH studies without early discontinuation may join. This study continues the observation and assessment of participants over an extended period to learn about the effects and safety of sotatercept combined with background PAH treatments. During the study, participants will be regularly monitored for adverse events, treatment discontinuations, and the presence of anti-drug antibodies for up to approximately 90 months. Laboratory tests will evaluate blood components such as platelets, hemoglobin, creatinine, bilirubin, and liver enzymes. Changes from baseline in body weight, blood pressure, and electrocardiogram readings will also be tracked. The study involves adherence to visit schedules and compliance with study procedures to ensure comprehensive long-term safety data collection.

Researchers are evaluating KK8123, a subcutaneous drug, in adults with X-linked hypophosphatemia (XLH) through a Phase 1/2, open-label, dose-escalation study. The trial aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of KK8123, including an optional safety extension. This study involves adults aged 18 to 65 years diagnosed with XLH, focusing on various laboratory and clinical measures over extended periods. The study begins with a Screening Period lasting up to 28 days, including informed consent. Part 1 is the Dose Escalation Period with a planned Treatment and Observation Period lasting 32 to 44 weeks. Participants receive subcutaneous doses of KK8123 during this time. Part 2 is an optional Extension Period lasting up to 52 weeks for participants who complete Part 1, continuing treatment and safety monitoring. Participants undergo regular assessments including blood tests for hematology, clinical chemistry, hormone levels, and serum phosphorus. Vital signs such as temperature, pulse, respiratory rate, and blood pressure are monitored. Imaging tests like echocardiograms and renal ultrasounds are performed. KK8123 drug levels are measured in the blood over time. Safety is tracked by recording treatment-emergent adverse events throughout the study. Participants are expected to comply with study visits, medication regimens, and contraception requirements during and after treatment.

Researchers are evaluating the long-term safety and effects of nerandomilast in people with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF) who have previously completed treatment with nerandomilast in earlier studies. The study aims to understand how well participants tolerate nerandomilast over time, and whether it helps improve lung function, delays symptom worsening, reduces hospital visits, or impacts survival. This is a Phase 3 open-label extension trial. Participants take nerandomilast tablets daily for up to 1 year and 10 months while continuing their usual pulmonary fibrosis treatments. The study follows an open-label design where all participants receive nerandomilast. There are no placebo or comparator groups in this extension phase. Throughout the study, participants regularly visit their doctors for health assessments and lung function tests. Doctors monitor any health problems or side effects experienced during treatment. The main outcome measured is whether participants experience any adverse events up to the final follow-up visit, which occurs at week 99. This close monitoring helps evaluate the long-term safety and potential benefits of nerandomilast in this patient group.

Researchers are evaluating the safety, tolerability, and effectiveness of two treatments, inebilizumab and blinatumomab, in adults with active and difficult-to-treat autoimmune diseases. This includes systemic lupus erythematosus (SLE) with nephritis, SLE with and without nephritis, and active refractory rheumatoid arthritis (RA). The study is a Phase 2, open-label, multicenter platform trial designed to assess these treatments across different subprotocols based on the specific condition and disease activity. Participants receive inebilizumab through intravenous infusion or blinatumomab via subcutaneous injection, depending on their assigned subprotocol. The study includes several parts: Subprotocol A focuses on SLE with nephritis treated with inebilizumab; Subprotocol B Part A and Part B assess blinatumomab in SLE with and without nephritis; and Subprotocol C Parts A and B evaluate blinatumomab in rheumatoid arthritis. The treatments are administered over specified periods, with some groups receiving treatment for up to 52 weeks. During the trial, participants undergo various assessments to monitor safety and disease response, including evaluation of treatment-emergent adverse events, serious adverse events, and measures of disease remission or activity. For example, kidney response and remission in SLE and disease activity scores in RA are measured at specific time points. Safety monitoring continues through the treatment period, with data collected on adverse events from Day 1 to Week 52. Participants' health status, laboratory tests, and disease activity are regularly evaluated to understand the treatments' effects and tolerability.

Researchers are investigating the effects of multiple doses of vosoritide and comparing its therapeutic dose to human growth hormone (hGH) in children with idiopathic short stature (ISS). This Phase 2 study aims to understand how these treatments influence growth in this population. After an initial observation period of at least 6 months to measure baseline growth, participants are randomly assigned to receive either vosoritide, placebo, or hGH (the latter only in the United States). Those in the vosoritide and placebo groups undergo up to 6 months of randomized treatment, followed by open-label vosoritide until they reach near-final adult height or at least age 16 for females or 18 for males. Participants assigned to hGH receive open-label treatment for a minimum of 4 years. Throughout the study, safety is carefully monitored with clinical and imaging assessments focused on hips and lower extremities, as well as watching for hypotension, fractures, and slipped capital femoral epiphysis. An independent Data Monitoring Committee reviews safety data regularly. Study visits include a treatment completion visit about 4 weeks after the last dose, and follow-up assessments may continue annually through the end of the study. Key outcome measures include changes in annualized growth velocity at 6 months and changes in height and height Z-score after 4 years.

Researchers are conducting a phase II trial to evaluate the clinical effectiveness, safety, and tolerability of MAS825 in children and adults diagnosed with Still's disease. The study focuses on participants with active disease signs such as elevated CRP or ferritin levels, fever, rash, arthritis, serositis, or macrophage activation syndrome, who also require glucocorticoid treatment. This trial aims to gather important information about MAS825's role in managing Still's disease across a wide age range. Participants will receive MAS825, an experimental drug, in an open-label format, meaning all participants will be given the study drug without a placebo comparison. The study does not specify dosing details but monitors participants closely for response and safety. There is no mention of separate treatment arms or extension periods in the provided information. Throughout the study, researchers will assess participants' clinical response by monitoring disease activity markers and symptoms up to Day 85. Safety and tolerability will also be evaluated through regular assessments. The trial includes pediatric and adult participants aged 1 to 100 years and tracks the number of participants showing a clinical response based on predefined criteria. Participants will be monitored for adverse events, and their need for glucocorticoids will be considered during the study.

Researchers are evaluating the safety and effectiveness of two doses of inhaled pirfenidone (called AP01) compared to a placebo in people with progressive pulmonary fibrosis (PPF). This Phase 2b study is randomized, double-blind, and placebo-controlled, involving up to 300 participants who will continue their standard care during the 52-week trial. The goal is to see how well AP01 works and how safe it is when added to usual treatments for PPF. Participants will be randomly assigned to one of three groups: high-dose AP01, low-dose AP01, or placebo. All treatments are given as an oral inhalation solution twice daily. The study will last for 52 weeks, during which researchers will monitor and compare the effects of these treatments on lung function and disease progression. During the study, participants will undergo various assessments including lung function tests and clinical evaluations to track their respiratory health. Researchers will check for changes in lung capacity and symptoms and monitor safety throughout the treatment period. The main outcome measured is the impact of AP01 doses compared to placebo after 52 weeks of treatment.

Researchers are evaluating the safety, effectiveness, and how the body processes and responds to atumelnant treatment in children with classic congenital adrenal hyperplasia (CAH), a genetic condition affecting hormone production. This Phase 2/3 study includes an open-label extension to continue monitoring participants over time. Approximately 153 children aged 1 to under 18 years are expected to join the study. The study is divided into three parts. Part A is an open-label Phase 2 study with sequential cohorts starting with ages 12 to under 18 years, followed by children aged 1 to 11 years after safety reviews. Part B is a Phase 3, double-blind, randomized, placebo-controlled part where participants receive either atumelnant or placebo tablets once daily, dosed according to weight. Part C is an open-label extension allowing participants from Parts A and B to continue treatment based on investigator decision. Participants will be monitored through blood tests measuring hormone levels, including morning serum androstenedione, and will have their glucocorticoid doses tracked over time. The study will assess changes in hormone levels at weeks 8, 28, and up to week 260. Safety, compliance with medication, thyroid function, and other health parameters will be regularly evaluated throughout the study. The total participation duration varies depending on the study part involved.

Researchers are evaluating the safety and effectiveness of the drug baricitinib in children aged 1 year to less than 18 years who have systemic juvenile idiopathic arthritis (sJIA), a type of arthritis that affects the joints and causes inflammation. This Phase 3 study includes two groups of participants: one group receives either baricitinib or tocilizumab, and the other group receives only baricitinib. The study aims to understand how well baricitinib works compared to tocilizumab and to monitor its safety in young patients. Participants in the study are assigned to one of two cohorts. Cohort 1 includes children who have not previously received IL-6 inhibitor therapy and will be treated with either baricitinib, taken by mouth, or tocilizumab, given by subcutaneous injection. Cohort 2 includes children who will receive baricitinib alone. The treatments are administered according to the study plan, and participants are monitored throughout the treatment period. During the study, researchers will assess participants' joint activity and overall response to treatment by measuring the percentage who meet specific improvement criteria after 12 weeks. Participants will undergo regular evaluations including physical exams and safety monitoring. The study also closely watches for any side effects or infections. The total duration of participation includes the treatment period and follow-up assessments to ensure comprehensive evaluation of baricitinib's effects in children with sJIA.