Brest

Researchers are evaluating the "Deneo Kid" digital application designed to improve coordination and quality of rehabilitation care for children and young people with neuromotor disabilities. This observational multicenter study focuses on assessing whether the app is easy to use, acceptable, and if it helps families and rehabilitation professionals provide more integrated, family-centered, and coordinated care. The study also examines the app's impact on communication, relationships, and the time and costs involved in care coordination. Participants include children, adolescents, and young people aged 2 to 25 years with neuromotor disabilities, their parents or legal guardians, and rehabilitation professionals involved in their care. Each participating team will use the secured "Deneo Kid" application for three months to share information, track care goals, and communicate. The study is conducted across four pediatric rehabilitation centers in France. Before and after using the app, participants complete surveys, and some take part in interviews or focus groups to share their experiences. During the study, researchers observe app usage in various rehabilitation settings and collect data on usability, acceptability, care coordination, user experience, and coordination costs and time. Usability is measured from enrollment through the three-month application test. Additional assessments include questionnaires on integrated care and family-centered care, interviews, focus groups, digital journals, and direct observations. The study aims to provide insights into the app’s potential for supporting better rehabilitation coordination and inform larger future studies.

Researchers are evaluating the potential use of the novel imaging agent 68Ga-FAPI46 in positron emission tomography combined with computed tomography (PET/CT) to improve diagnosis and monitoring of various chronic inflammatory and fibrosing diseases. This single-center pilot study focuses on 13 different medical conditions, including rheumatoid arthritis, liver fibrosis, and systemic lupus, aiming to explore how this imaging method might enhance disease assessment and guide future clinical development. The study uses the 68Ga-FAPI46 radiotracer, which targets Fibroblast Activation Protein (FAP) found on activated fibroblasts involved in disease processes. Depending on the clinical situation, participants will undergo a baseline 68Ga-FAPI PET/CT scan, with some patients receiving additional scans at 3 and 6 months if their therapeutic management is intensified at inclusion. The imaging is planned either as a one-time evaluation or as a longitudinal assessment to track disease changes over time. Participants will be asked to undergo these PET/CT scans and provide informed consent before joining. Researchers will measure the intensity of PET tracer uptake at the start of the study to evaluate diagnostic usefulness. The study includes safety monitoring and may involve follow-up imaging for those with changing treatments, helping to determine the value of 68Ga-FAPI46 PET/CT in routine care for these chronic conditions.

Researchers are evaluating treatments for breast cancer that is hormone receptor-positive (HR+) and human epidermal growth factor receptor 2-negative (HER2-), specifically in cases where the cancer is either locally advanced and cannot be removed by surgery or has spread to other parts of the body (metastatic). The study aims to determine if patritumab deruxtecan (also called HER3-DXd or MK-1022) helps patients live longer overall or without the cancer growing compared to chemotherapy or trastuzumab deruxtecan. This is a Phase 3 clinical trial focusing on this particular type of breast cancer. Participants receive one of several treatments: patritumab deruxtecan through intravenous infusion, chemotherapy options like paclitaxel or nab-paclitaxel via IV, oral capecitabine tablets, liposomal doxorubicin via IV, or trastuzumab deruxtecan via IV infusion. The study compares the effects of patritumab deruxtecan alone to the treatment chosen by the physician. Treatments are administered according to standard dosing schedules during the trial. During the study, participants are monitored for how long they live without the cancer progressing (up to about 45 months) and overall survival (up to about 85 months). Researchers assess disease status through imaging and other evaluations. Participants have regular check-ups to monitor health, treatment effects, and any side effects. The study tracks treatment response and safety over the extended follow-up period to understand the benefits and risks of the therapies.

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 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 long-term safety and tolerability of dazodalibep in adults with Sjögren's Syndrome. This phase 3 open-label extension study focuses on participants who have previously received dazodalibep or placebo in earlier phase 3 trials and completed those studies through Week 48. Participants will receive dazodalibep intravenously during this long-term extension study. The first dose is administered around Week 48 (+28 days) following the prior phase 3 studies. The study monitors safety and tolerability over an extended period to assess treatment-emergent adverse events up to 152 weeks. During the study, participants will undergo regular evaluations to monitor their health and any side effects. Researchers will collect data on adverse events that emerge during treatment. The overall goal is to gather long-term safety information to better understand how participants tolerate dazodalibep when used over an extended time frame.

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 the drug STP938 in adults with high risk essential thrombocythaemia (ET) who have not responded well to or cannot tolerate hydroxycarbamide therapy. This phase 1b, open-label study aims to determine if STP938 can control platelet counts effectively and safely without causing unwanted side effects. The study focuses on patients who require treatment to lower their platelet counts due to high risk ET. Participants will take STP938 capsules daily in 28-day cycles for about 12 months. The initial dose will be assigned for the first 4 weeks and may be adjusted by the investigator as needed. Study visits will occur approximately twice per cycle, totaling about 26 visits over the year. If the drug controls platelet counts without significant side effects, participants may continue treatment beyond 12 months. During the study, participants will have physical exams, ECGs, blood and urine tests, CT or MRI scans, bone marrow biopsies, drug level testing, and gene testing. They will also complete monthly symptom questionnaires. After treatment ends, safety follow-up visits will ensure no adverse effects remain. The main outcomes measured are clinical efficacy and safety over approximately 12 months of participation.

Researchers are evaluating the effectiveness, safety, and tolerability of two different doses of remibrutinib compared to a placebo in adults and adolescents with moderate to severe hidradenitis suppurativa (HS). This phase 3 study aims to determine how well remibrutinib works in treating this chronic skin condition characterized by painful abscesses and inflammatory nodules. The study lasts a total of 76 weeks and includes several phases: up to 4 weeks for screening, followed by a 16-week double-blind treatment period where participants receive either remibrutinib Dose A, Dose B, or a matching placebo. After this, there is a 52-week treatment period where all participants receive remibrutinib (Dose A or Dose B). Finally, a 4-week safety follow-up period occurs without treatment. Participants who stop treatment early are encouraged to stay in the study and complete the safety follow-up. During the study, participants will be regularly assessed for clinical response to treatment, focusing on the proportion achieving a 50% improvement in HS symptoms by week 16. Researchers will monitor safety and tolerability throughout the study, including during the follow-up period. Various evaluations such as physical exams and clinical assessments will be conducted to measure treatment effects and ensure participant safety over the entire 76-week duration.

Researchers are investigating the effectiveness, safety, and tolerability of combining baxdrostat with dapagliflozin compared to dapagliflozin alone in people with chronic kidney disease (CKD) and high blood pressure. This Phase III, international, multicenter, double-blind, placebo-controlled study aims to see if this combination reduces risks such as significant kidney function decline, kidney failure, heart failure events, or cardiovascular death. The study includes a 4-week run-in period where participants not previously treated with SGLT2 inhibitors receive dapagliflozin alone. After this, participants are randomly assigned to receive either baxdrostat plus dapagliflozin or placebo plus dapagliflozin in a double-blinded manner. Study visits occur frequently initially (at 2, 4, 8, 16, 34, and 52 weeks after randomization) and then approximately every 4 months. If participants stop the blinded treatment early, they continue dapagliflozin alone unless specific criteria require its discontinuation. Participants will undergo regular assessments including blood pressure monitoring and laboratory tests related to kidney function and cardiovascular health. The primary outcome measures the reduction in risk of major kidney and heart events over up to 37 months. Even if participants stop the study treatment, they will continue follow-up visits and data collection to ensure comprehensive safety and efficacy evaluation throughout the study duration.