Baracaldo

This trial evaluates the effectiveness of adjuvant cemiplimab immunotherapy in adults with surgically removed stage II-IIIA non-small cell lung cancer NSCLC who have not previously received adjuvant platinum-based chemotherapy. The study aims to compare disease-free survival between patients treated with cemiplimab and those under observation without additional treatment, focusing on patients with tumors showing PD-L1 expression of 1% or higher. Participants are randomly assigned to receive either cemiplimab or observation. Those in the cemiplimab group receive 350 mg intravenously every 3 weeks for 4 cycles, followed by 700 mg every 6 weeks for up to 6 cycles or until disease relapse or unacceptable side effects occur. The observation group does not receive adjuvant treatment. Treatment continues until relapse, toxicity, or completion of planned cycles. During the trial, participants undergo regular assessments including imaging and laboratory tests to monitor disease status and side effects. The main outcome measured is disease-free survival, tracked for approximately 59 months from randomization. Secondary outcomes include overall survival and the nature and severity of adverse events. Participants are monitored for safety and treatment response throughout the study period, which is expected to conclude in March 2029.

Researchers are investigating new treatments for people with high-risk, localized non-small cell lung cancer NSCLC that has been completely removed by surgery. The study aims to find out if giving one or two specific treatments after surgery can help prevent the cancer from returning. This Phase 3 trial focuses on participants with Stage I NSCLC who have certain high-risk features. Participants are randomly assigned to one of three groups one group receives intismeran combined with pembrolizumab coformulated with berahyaluronidase alfa, another group receives intismeran alone, and a third group receives a placebo. Intismeran is given as an intramuscular injection, while pembrolizumab with berahyaluronidase alfa is administered subcutaneously. The trial compares disease-free survival among these groups over an extended period. Throughout the study, participants will be monitored regularly with health assessments and questionnaires to evaluate quality of life and physical functioning. Researchers will track how long participants remain free from cancer and observe any side effects or adverse events. The study may last up to several years, with ongoing safety and outcome evaluations to better understand the treatments impact.

Researchers are evaluating NEU-411 in adults aged 40 to 80 years who have early Parkinsons Disease PD with elevated activity in the LRRK2 pathway, identified through a genetic test. This Phase 2 study aims to assess the safety and effectiveness of NEU-411, a selective brain-penetrant inhibitor of LRRK2, compared to placebo in participants with LRRK2-driven PD. Participants will be randomly assigned to receive either NEU-411 at 30 mg once daily or a matching placebo for a 52-week treatment period. After this phase, participants may join an open-label extension to receive NEU-411 treatment for an additional 26 weeks. The study evaluates treatment effects on PD symptoms and adverse events. During the study, participants will undergo assessments including the Roche digital biomarker score using a Parkinsons Disease application and the Movement Disorder Societys Unified Parkinsons Disease Rating Scale. Safety monitoring includes tracking treatment-emergent and serious adverse events, with a follow-up visit scheduled within two weeks after treatment ends. Total study participation may extend up to 78 weeks including the extension phase.

Researchers are evaluating the safety and effects of a medicine called fosmanogepix for treating candidemia and invasive candidiasis, which are serious fungal infections caused by Candida yeast. This Phase 3 clinical trial compares fosmanogepix to the standard treatment using caspofungin followed by fluconazole, aiming to show that fosmanogepix is not worse than the standard treatment by a margin of 15%. The study includes adult patients diagnosed with these infections and is sponsored by Basilea Pharmaceutica. Participants are randomly assigned to one of two groups two-thirds receive fosmanogepix intravenously, with an option to switch to oral tablets, while one-third receive caspofungin intravenously followed by oral fluconazole. Matching placebos are given to maintain blinding. Treatments are given daily, first by IV infusion at the clinic and then orally either at the clinic or at home if discharged. Treatment duration can be up to six weeks, depending on infection clearance and symptom improvement. Participants will be monitored through multiple study visits, with assessments including survival status at 30 days, treatment success at the end of treatment, and follow-up evaluations six weeks after stopping treatment. Additional evaluations include clinical and mycological responses, blood cultures, safety monitoring such as adverse events, lab tests, neurological exams, ECGs, and drug concentration measurements. The total study duration for each participant may be approximately 12.5 weeks, considering treatment and follow-up periods.

Researchers are evaluating the efficacy and safety of fosmanogepix, given either intravenously or orally, for treating adult patients diagnosed with invasive mold infections. This Phase 3 trial compares fosmanogepix to the standard antifungal therapies. The study mainly aims to assess all-cause mortality by Day 42 and includes patients both receiving primary therapy and those receiving salvage treatment after prior therapies failed or were not tolerated. Participants are assigned to one of two cohorts Cohort A, where patients receive either fosmanogepix or the best available standard antifungal treatment, and Cohort B, where patients receive only fosmanogepix as salvage therapy. Fosmanogepix is administered via IV infusion or oral tablets. The treatment phase targets 84 days but can be extended up to 180 days, followed by a follow-up period. During the study, participants undergo various assessments including mortality evaluation at Day 42, clinical and radiological response checks, laboratory tests, neurological exams, ECG monitoring, and plasma drug level measurements at multiple time points. Safety is closely monitored throughout the study and follow-up, which together may last approximately 8 months. Researchers will track adverse events and overall treatment success over this period.

Researchers are evaluating the clinical efficacy, safety, and tolerability of XEN1101 as an additional treatment for people with focal-onset seizures in a Phase 3 randomized, double-blind, placebo-controlled study. This trial aims to compare two doses of XEN1101 with a placebo to see how well the medication can reduce seizure frequency in patients who continue their current antiseizure medications. The study involves adults diagnosed with focal epilepsy who have tried at least two antiseizure medicines without achieving seizure freedom. About 360 participants will be randomly assigned to receive either 25 mg or 15 mg of XEN1101 or a placebo once daily with an evening meal. The study includes up to 9.5 weeks of baseline monitoring to track seizure frequency followed by 12 weeks of blinded treatment. Participants maintaining the study drug can then join an open-label extension to continue treatment or enter an 8-week follow-up after treatment ends. Throughout the study, participants will keep accurate seizure diaries and continue their stable antiseizure medications. Researchers will measure the median percentage change in seizure frequency from baseline through the 12-week treatment period, along with secondary outcomes like the proportion of participants with at least a 50% reduction in seizures and patient-reported improvement. Safety will be monitored from screening until 56 days after the last dose. Overall, participants are involved for the baseline, treatment, and follow-up phases lasting several months.

This research aims to observe the real-world use and effects of pegcetacoplan in adults diagnosed with Paroxysmal Nocturnal Hemoglobinuria PNH. As a new treatment with a unique mechanism of action, pegcetacoplans effectiveness and safety in routine medical practice are being studied to provide valuable information for patients, healthcare providers, and payers. The study will also gather data on blood transfusions and healthcare resource use before and after starting pegcetacoplan. Patients who have started pegcetacoplan treatment within the past 12 months or are prescribed the drug at enrollment will be included. Data collection includes retrospective information from up to 12 months before treatment start and prospective monitoring for approximately 36 months, with the total data period extending up to about 48 months. After stopping pegcetacoplan, patients remain in the study for 8 weeks to record any adverse events. Patients continue regular clinic visits, where data from each visit will be gathered. Participants will have data collected on various health measures such as hemoglobin levels, blood markers, transfusion needs, and patient and physician treatment satisfaction at regular intervals up to 36 months. Safety and adverse events will be monitored throughout. The main outcome measured is the change in hemoglobin level from treatment start to 6 months. This long-term observational study allows for comprehensive tracking of pegcetacoplans use and effects over time in usual care settings.

Researchers are evaluating the long-term safety and effects of etavopivat, a new oral medicine being developed for inherited blood disorders such as sickle cell disease and thalassaemia. These conditions affect haemoglobin, the protein responsible for carrying oxygen in the blood. This phase 3 study involves participants who have already completed treatment in a prior etavopivat study and aims to understand how etavopivat performs over an extended period of up to 264 weeks, though the study may end earlier if the drug gains approval locally. Participants will receive oral doses of etavopivat, with different forms A, B, or C given based on their age and condition. Those aged 12 years and older will receive etavopivat A or C, while children under 12 years will receive etavopivat B. The study includes several groups covering various sickle cell disease and thalassaemia categories, including transfusion-dependent and non-transfusion-dependent cases. Treatment is continuous during the study period. Throughout the study, participants will undergo regular monitoring for side effects and treatment responses, including tracking treatment emergent adverse events, hospitalizations, vaso-occlusive crises, hemoglobin concentration changes, and blood transfusion needs. These assessments will help evaluate the drugs safety and efficacy across age groups and disease types. The study is open-label and non-randomized, with follow-up lasting up to about six years.

This research aims to describe the characteristics, clinical outcomes, and event rates in participants with propionic acidemia PA, a rare metabolic disorder. It is a non-interventional, observational, global, multicenter study that collects data retrospectively from medical records to better understand PA over time. Data will be gathered by reviewing medical records from various study sites, including hospitals, clinics, and academic centers. Participants included will be those diagnosed with PA confirmed by genetic testing, with records documenting specific metabolic events. The study will cover up to 10 years of medical history and events. Participants involvement consists of allowing researchers to review their medical records to collect information about metabolic decompensation events, hospitalizations, and urgent healthcare visits related to PA. Researchers will analyze the number and severity of these events, along with hospitalization data, to understand PAs clinical impact. The study does not involve direct treatment or interventions and focuses on data collection and analysis.

Researchers are evaluating the effects of a triple therapy inhaler combining budesonide, glycopyrronium, and formoterol fumarate BGF MDI 32014.49.6 g compared to a dual therapy inhaler with glycopyrronium and formoterol fumarate GFF MDI 14.49.6 g on heart and lung outcomes in adults with Chronic Obstructive Pulmonary Disease COPD who have a higher risk for heart and lung events. This Phase III study is randomized, double-blind, and conducted at multiple centers, focusing on participants with COPD and elevated cardiopulmonary risk. Participants will receive either the triple therapy inhaler or the dual therapy inhaler, both administered twice daily. The study compares these two inhalers over a period of up to three years, monitoring for serious cardiac or COPD events. The trial includes careful evaluation of various heart and lung-related health events during this period. During the study, participants will be closely monitored through regular visits, assessments, and tests to measure lung function, heart events, and COPD exacerbations. Researchers will track the time until the first severe cardiac or COPD event and evaluate other cardiovascular and respiratory outcomes over up to three years. Participants will also be assessed for their ability to properly use the inhaler and adherence to the study protocol throughout the trial.