Esplugues De Llobregat

Researchers are evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of a drug called S230815 in children aged 2 to 12 years who have Developmental Epileptic Encephalopathy caused by specific genetic changes in the KCNT1 gene. This is a Phase Ib/II, first-in-human, multicenter, open-label study focusing on this rare and severe form of epilepsy. The study includes two main parts. Part 1 involves giving participants increasing doses of S230815 through injection to determine the best dose. After completing Part 1, participants may enter Part 2, a long-term extension where they continue receiving their assigned dose of S230815 for up to 72 weeks. Participants will be closely monitored throughout the study for side effects and drug levels. Researchers will collect safety information, including any adverse events, for up to 116 weeks. Genetic testing confirms eligibility, and participants will undergo various assessments during the screening and treatment periods to evaluate the drug's effects and safety over time.

This research investigates the long-term effects of mirikizumab in children and adolescents aged 2 to 19 years with moderate-to-severe ulcerative colitis or Crohn's disease. The study is designed as a Phase 3, multicenter, open-label extension trial aiming to assess the ongoing safety and efficacy of this treatment in pediatric participants. It includes those who have completed previous related studies and are expected to benefit from continued mirikizumab treatment. Participants will receive mirikizumab either by subcutaneous injection or intravenous infusion as part of this extended treatment. The study may last approximately 172 weeks and involve up to 44 visits over this period. There is also a possibility for participants to continue receiving treatment through a Continued Access Period after the main study. Throughout the study, participants will be regularly monitored with clinical assessments to determine remission status using the Modified Mayo Score for ulcerative colitis and the Pediatric Crohn's Disease Activity Index for Crohn's disease at week 52. Safety and efficacy will be closely followed, including the evaluation of any adverse events or changes in disease activity, ensuring comprehensive long-term observation.

Researchers are conducting a master protocol study called CAMPFIRE to efficiently carry out multiple clinical trials testing new drugs in children and young adults with cancer. This master protocol allows for adding new studies as new cancer drugs become available, focusing on the treatment of measurable or evaluable tumors in participants aged 1 to 39 years. The goal is to evaluate various drugs under a unified research plan to improve treatment options for young cancer patients. The study involves several investigational drugs administered either intravenously or orally, including Ramucirumab, Cyclophosphamide, Vinorelbine, Gemcitabine, Docetaxel, Abemaciclib, Irinotecan, and Temozolomide. Each drug is tested under specific clinical trials within the master protocol, with treatment schedules and dosing tailored to each drug. Participants receive these treatments following standard clinical procedures, with adjustments based on individual study protocols and treatment responses. Participants will be closely monitored throughout the trial, with assessments including performance status evaluations, laboratory tests to check organ and blood function, and pregnancy testing for females of childbearing potential. Researchers will track how many participants receive each treatment during the first four weeks and observe the duration of treatment benefits. Safety evaluations, adherence to contraceptive measures, and recovery from prior therapies are also part of the study monitoring. Participation duration and additional assessments depend on the specific trial and treatment plan assigned.

Researchers are evaluating the efficacy and safety of benralizumab, given as a subcutaneous injection, in children and adolescents aged 6 to under 18 years who have severe eosinophilic asthma. These patients have a history of asthma exacerbations and uncontrolled symptoms despite treatment with high-dose inhaled corticosteroids plus at least one other controller medication. This Phase III study aims to compare benralizumab to placebo in reducing the time to the first asthma exacerbation. The study includes a screening period lasting from 4 to 12 weeks to confirm eligibility. After screening, patients are randomly assigned in a 1:1 ratio to receive either benralizumab or placebo via subcutaneous injections during a double-blind treatment period lasting a minimum of 16 weeks. This period continues until the patient experiences an asthma exacerbation or a set number of events occur. Patients who exacerbate can enter an open-label extension where all receive benralizumab for at least 48 weeks. An end-of-treatment visit occurs 8 weeks after the last dose in the extension phase. Participants will be monitored through visits and assessments including confirmation of severe eosinophilic asthma, asthma control questionnaires, and symptom diaries. Researchers will measure the time to first asthma exacerbation as the primary outcome. Medication adherence is tracked during screening, and safety is monitored throughout both the double-blind and extension periods. Total participation may span over a year, considering screening, treatment, extension, and follow-up visits.

Researchers are studying participants with Congenital Myasthenic Syndromes (CMS) caused by mutations in the DOK7, MUSK, AGRN, or LRP4 genes. The goal is to understand symptoms, quality of life, and disease activity in these patients over time. This natural history study gathers important information about how CMS affects participants and tracks changes in their health. Participants will attend up to four study visits during which clinical assessments are performed. These assessments focus on evaluating symptoms and overall quality of life related to CMS. No specific treatments or interventions are administered, as the study is observational and aims to collect detailed data about the condition's progression. Throughout the study, researchers collect data retrospectively and prospectively over a period of up to 12 months. This includes information on diagnosis, healthcare use, medications, and changes in health status related to CMS. The study carefully monitors participants to better characterize the natural course of the disease and its impact on daily living.

Researchers are evaluating CRD-4730 in a Phase 2 clinical trial to study its safety, tolerability, pharmacokinetics, and pharmacodynamics in people with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). This condition is confirmed by genetic testing and clinical symptoms. The trial is multicenter, double-blind, and placebo-controlled, involving repeated doses of the study drug. Participants will join a 3-period, randomized, crossover study where each person receives two different doses of CRD-4730 and one matching placebo dose in varying sequences. CRD-4730 is given orally as tablets, and placebo tablets match the drug tablets in appearance. The study carefully compares the effects of these treatments over the course of the trial. During the study, participants will undergo exercise stress tests to monitor heart activity and frequent evaluations to assess safety and drug effects. Researchers will measure outcomes from the start of treatment to Day 101. Safety monitoring includes checking heart rhythms, blood pressure, liver function, and other health indicators. Participants will be followed closely to observe how they respond to the treatments throughout the study duration.

The total study duration per patient will be 166 weeks that will consist of a 4- week screening, a 12-week core treatment phase, a 144-week extension phase, and a 6-week post-treatment follow-up.

Researchers are evaluating how well two new study drugs, CagriSema and cagrilintide, help children and adolescents with excess body weight lose weight. This trial includes participants aged 8 to less than 18 years who have overweight or obesity. The study is designed in two parts: a main study and an extension study. The main study compares CagriSema, cagrilintide, semaglutide (an already approved drug), and placebo, with treatments assigned randomly. Participants receiving semaglutide will not continue to the extension study. The total time in the main study is about 1 year and 6 months, while those in the extension study may participate for up to about 4 years and 10 months. Participants in the main study will receive one of the four treatments by subcutaneous injection. In the extension study, participants will receive either CagriSema or cagrilintide. The study drugs are monitored closely for safety, and participants may experience side effects. The study compares these new treatments to a placebo and an existing approved drug to better understand their effects on weight management in young people. During the study, researchers will measure changes in body mass index (BMI) from baseline to week 68 as the primary outcome. Participants will undergo various assessments including laboratory tests and physical evaluations. The study tracks adherence to treatment and monitors safety throughout the study period. This comprehensive approach aims to provide detailed information about the efficacy and safety of these medications for managing weight in children and adolescents.

Researchers are conducting a non-interventional, observational, retrospective study to describe the characteristics, clinical outcomes, and event rates of participants diagnosed with propionic acidemia (PA). This global, multicenter study focuses on gathering medical record data from participants who meet specific inclusion criteria related to PA diagnosis and clinical history. Medical data will be collected through review of records from medical clinics, hospitals, and academic medical centers. The study includes participants with confirmed PA diagnosis based on molecular genetic testing of specific enzyme subunits. Data must support clinical event adjudication and cover participants' history from birth or January 1, 2015, onward. The study records metabolic decompensation events (MDEs) experienced by participants over up to 10 years. Participant involvement consists of medical record abstraction without intervention or direct treatment. Researchers will evaluate the number of MDEs adjudicated by a clinical event committee. Eligibility requires informed consent and sufficient data availability. Participants with certain prior treatments or conditions, such as gene therapy, organ transplantation, or participation in specific clinical trials, will be excluded. The observation period includes retrospective data spanning multiple years to assess long-term clinical events and outcomes.

This research aims to evaluate the effectiveness and safety of an oral drug called ozanimod (RPC1063) in treating children and teenagers aged 2 to 17 years who have moderate to severe active ulcerative colitis (UC). These young participants have not responded well to standard treatments, and the study focuses on helping them achieve and maintain clinical remission of their condition. Participants will receive specified doses of ozanimod by mouth according to a set schedule. The study is a Phase 2/3, randomized, double-blind trial conducted at multiple centers. The goal is to assess how well ozanimod works and how safe it is in this pediatric population with UC that extends beyond the rectum. During the study, researchers will monitor participants closely through medical assessments, including endoscopy to confirm disease extent and other evaluations to track disease remission. The main outcome measured is the proportion of participants who reach clinical remission by Week 52. Safety and drug behavior in the body will also be observed throughout the trial period.