Esplugues De Llobregat

Researchers are studying pediatric participants aged 2 to 12 years with Developmental Epileptic Encephalopathy caused by a genetic variant in the KCNT1 gene. This Phase IbII, first-in-human, multicenter, open-label study aims to assess the safety, tolerability, and how the investigational drug S230815 behaves in the body and affects the disease. The trial is sponsored by Institut de Recherches Internationales Servier and includes a screening period followed by two treatment parts. Participants will receive multiple ascending doses of S230815 administered intrathecally during Part 1 of the study. Those who complete Part 1 may continue into Part 2, a long-term treatment extension lasting up to 72 weeks, continuing with the same dose cohort as in Part 1. The treatment is given as a solution for injection, and dosing is designed to evaluate safety and drug effects over time. Throughout the study, participants will be closely monitored for adverse events and drug levels in cerebrospinal fluid and plasma. Seizure frequency will be tracked using daily logs and periodic 24-hour video EEG assessments. Researchers will also record the use of rescue medications. The total participation may last up to 116 weeks, including the screening, treatment, and follow-up visits, ensuring thorough evaluation of the drugs impact and safety profile.

Researchers are evaluating the long-term effects of mirikizumab treatment in children and teenagers aged 2 to 19 years with moderate-to-severe ulcerative colitis or Crohns disease. This phase 3 study aims to assess the clinical remission rates and other health outcomes related to these conditions over an extended period. The study is sponsored by Eli Lilly and Company and follows a treatment focus for pediatric participants with these inflammatory bowel diseases. Participants receive mirikizumab administered by subcutaneous injections, with doses adjusted based on their weight. There are up to six planned doses, and if needed, intravenous rescue dosing is available if a participants condition worsens. The study may include a continued access period providing additional treatment beyond the main study duration. Participants are involved for about 172 weeks, attending up to 44 visits throughout the study. Regular assessments include evaluating clinical remission using the Modified Mayo Score for ulcerative colitis and the Pediatric Crohns Disease Activity Index for Crohns disease, along with other response and remission measures. Researchers monitor laboratory tests such as C-reactive protein levels and track corticosteroid use. Safety and health status are closely observed during the study and any continued treatment periods.

Researchers are conducting a master protocol trial designed to efficiently study several different drugs for children and young adults with various types of cancer. This approach allows multiple clinical trials under one common research plan, with each trial focusing on specific cancers like desmoplastic small round cell tumor, synovial sarcoma, and Ewings sarcoma. New drug studies may be added over time as new treatments emerge. Participation depends on how long the treatment benefits last. The study evaluates combinations of drugs given in cycles, including intravenous and oral medicines such as ramucirumab, cyclophosphamide, vinorelbine, gemcitabine, docetaxel, abemaciclib, irinotecan, and temozolomide. Different treatment groups receive specific drug combinations tailored to the cancer type, with treatment cycles lasting 21 or 28 days depending on the regimen. Each group is randomized to receive either an experimental drug combination or an active comparator without any blinding. Participants will be involved from screening through treatment and monitored regularly. Researchers will assess how many participants are assigned to each specific intervention plan within the first four weeks and track treatment effects. Participants must have measurable or evaluable disease, adequate organ function, and meet performance status criteria. Female participants of childbearing potential undergo pregnancy testing and must use contraception during and after treatment. Safety, adherence, and side effects are carefully monitored throughout the study, which is expected to continue until 2027.

This research aims to better understand the natural history of Congenital Myasthenic Syndromes CMS caused by mutations in the DOK7, MUSK, AGRN, or LRP4 genes. It evaluates disease activity by collecting clinical data and quality of life information from participants with these specific genetic mutations. The study is observational and involves participants aged 2 years and older diagnosed with CMS due to these mutations. Participants will attend up to four study visits where clinical assessments will be performed. These assessments include evaluating symptoms and quality of life using tools like the Quantitative Myasthenia Gravis QMG score and other standardized measures. The study collects both retrospective and prospective data on diagnosis, healthcare use, medications, and health status changes related to CMS over a period of up to 12 months. During the study, participants will undergo various evaluations including symptom scoring, questionnaires on daily living activities, fatigue, and health-related quality of life. Researchers will analyze changes from baseline in these measures to understand disease progression and impact. No investigational treatments are given, and the focus is on monitoring and documenting the condition. Participation may last up to 12 months with scheduled visits for data collection and assessments.

Researchers are evaluating CRD-4730, an oral drug, in a Phase 2 clinical trial involving adults with Catecholaminergic Polymorphic Ventricular Tachycardia CPVT. This study aims to assess the safety, tolerability, pharmacokinetics PK, and pharmacodynamics PD of CRD-4730. Participants will be part of a randomized, double-blind, placebo-controlled crossover study to better understand how CRD-4730 affects CPVT symptoms and treatment responses. Participants will be randomly assigned to one of three sequences in a 3-period crossover design. Each participant will receive two different doses of CRD-4730 and one matching placebo dose in a random order. The study includes multiple dosing periods where participants take tablets of CRD-4730 or placebo, allowing researchers to compare the effects and tolerability of different doses against placebo. During the trial, participants will undergo various assessments to monitor safety, drug levels, and effects on their condition from baseline to Day 101. The study involves close monitoring of heart function and other health measures, with outcome evaluations occurring at multiple timepoints. This will help researchers understand how the drug behaves in the body and its impact on CPVT symptoms, with the trial lasting several months to complete all study periods and follow-ups.

Researchers are evaluating the drug LP352 in a phase 3, randomized, double-blind, placebo-controlled trial to study its effects on seizures in children and adults with Dravet Syndrome DS. This serious condition involves various seizure types with onset between 1 and 20 months of age. The study aims to test the efficacy, safety, and tolerability of LP352 compared to placebo over a total duration of about 24 months. Participants will be randomly assigned to receive either LP352 or a matching placebo. LP352 or placebo will be given orally or through a feeding tube. The study includes three main phases a Screening phase, a Titration period where doses are gradually increased to the highest tolerated level, and a Maintenance period to assess ongoing treatment effects. Afterward, participants will undergo a Taper period to reduce dosing and a Follow-Up phase for observation. During the study, participants will be monitored for seizure frequency changes, safety, and tolerability. Researchers will track countable motor seizures and measure percent change compared to baseline over up to 15 weeks. Participants or caregivers will complete seizure diaries, and stable antiseizure medication use is required. Safety evaluations will continue up to 21 weeks, with study visits scheduled throughout these phases. Total participation lasts approximately two years.

Researchers are conducting a phase II trial to evaluate the clinical benefit, safety, and tolerability of MAS825 arumakimig in both children and adults diagnosed with Stills disease who have not responded adequately to IL-1 andor IL-6 or other available treatments. The study also includes participants with associated lung disease or macrophage activation syndrome. This trial is open-label and single-arm, aiming to assess MAS825 in this patient group. Participants will receive the experimental drug MAS825 during two treatment periods following an initial screening phase. The total study duration for each participant is approximately 16 months. The trial is designed with three periods Screening, Treatment Period 1, and Treatment Period 2. MAS825 administration is the focus throughout the treatment periods, with no placebo or comparator groups. During the study, participants will undergo assessments including clinical response evaluations based on specific criteria by Day 85, biomarker level measurements, physician assessments, and quality of life evaluations. Researchers will monitor changes in glucocorticoid dosage and disease activity over time. Safety and tolerability are closely followed, and inactive disease status while on low-dose corticosteroids will be assessed by 15 months. The study is sponsored by Novartis Pharmaceuticals and includes both pediatric and adult participants aged 1 to 100 years.

Researchers are studying the use of sarilumab, a drug given by injection, in children and adolescents aged 1 to 17 years who have systemic juvenile idiopathic arthritis sJIA. The study aims to understand how the drug behaves in the body, its effects, and its long-term safety for treating this condition. This trial is a phase 2, open-label study sponsored by Sanofi, designed to find the best dose and treatment schedule for young patients with sJIA. Participants will receive sarilumab injections under the skin at doses that increase during the study based on body weight. The treatment includes a 12-week core phase where patients receive the drug, followed by a 144-week extension phase for continued treatment. After completing treatment, a 6-week follow-up period will monitor patients. The study includes careful dose adjustments and long-term observation to assess the drugs impact. Throughout the study, participants will undergo various assessments including blood tests to measure drug levels, evaluations of disease activity using scales like the Investigator Global Assessment and ParentPatient Global Assessment, and tracking of symptoms and medication use. Safety will be monitored continuously by recording any side effects or local reactions to injections. The total participation time is about 166 weeks, during which the researchers will collect data on how well sarilumab works and how safe it is for children and adolescents with sJIA.

Researchers are evaluating Mim8, a new medicine designed to help people with haemophilia A, including those with or without inhibitors. Mim8 aims to prevent bleeding episodes by replacing the function of the missing clotting factor VIII. This long-term study will last up to 5.5 years, ending either when Mim8 is approved in the participants country or by June 2028, whichever comes first. The study includes participants who have been involved in earlier related studies or are new infants with severe haemophilia A. Participants will receive Mim8 as a preventive treatment through subcutaneous injections. Depending on their entry point, participants may use an enhanced cartridge or a DV3407 pen-injector device for administering Mim8. The treatment is given regularly over the study period, with participants potentially receiving up to 262 injections. In the event of bleeding, additional haemostatic medications may be used as agreed with the study doctor. Female participants who are pregnant, breastfeeding, or planning pregnancy during the study are not eligible. During the study, participants will be monitored for any side effects, including injection site reactions and the development of antibodies against Mim8. Researchers will also track bleeding episodes, Mim8 blood levels, and device handling for some participants. Participants and their representatives will complete diaries and questionnaires about their treatment and health. Safety will be carefully followed throughout the study, which may last several years depending on individual enrollment and study progress.

This research aims to evaluate how well two new drugs, CagriSema and cagrilintide, help children and adolescents with excess body weight lose weight. The study includes participants aged 8 to under 18 years who have overweight or obesity. It is a Phase 3 trial that compares these new drugs with semaglutide, a drug already prescribed for weight management, and a placebo to understand their effects on weight loss. Participants in the main study are randomly assigned to receive one of four treatments CagriSema, cagrilintide, semaglutide, or placebo. All treatments are given once weekly as subcutaneous injections, starting with a dose escalation phase lasting up to 16 weeks, followed by a maintenance phase for 52 weeks. Those who receive semaglutide do not join the extension study. Participants in the extension study continue treatment with either CagriSema or cagrilintide for up to 156 weeks, while placebo participants follow a specific dosing regimen before continuing in the extension. During the study, participants will be monitored for changes in body mass index BMI and weight over time, with assessments at baseline, week 68, and for some measures, week 224. Researchers will also track body composition, metabolic markers, quality of life, and safety events. The entire duration for participants can be up to nearly five years if they take part in both the main and extension studies, involving regular visits and evaluations to understand the treatments effects and safety.