Baracaldo

Researchers are conducting a phase III randomized trial to evaluate the effectiveness of adjuvant cemiplimab immunotherapy in patients with surgically removed stage II-IIIA non-small cell lung cancer (NSCLC) who have not received prior platinum-based chemotherapy. The study focuses on patients whose tumors express PD-L1 at 1% or higher. The main goal is to measure disease-free survival, tracking the length of time patients remain free from cancer recurrence or death for up to about 59 months. Participants receive cemiplimab intravenously at 350 mg every 3 weeks for four cycles, followed by 700 mg every 6 weeks for six cycles, continuing until cancer relapse or unacceptable side effects occur. The treatment is compared to observation without additional adjuvant therapy. The study involves careful dosing schedules and monitoring to assess the impact of cemiplimab as a post-surgical treatment option. During the study, participants undergo brain imaging for staging, tumor PD-L1 testing, and regular pregnancy tests for women of childbearing potential. Researchers monitor disease recurrence and adverse effects throughout treatment and follow-up. Patients must meet specific health criteria, including recovery from surgery and adequate organ function, and agree to use contraception during treatment and for four months afterward. The total participation period includes treatment cycles and long-term follow-up to evaluate disease-free survival and safety.

Researchers are studying the effects of NEU-411 in men and women aged 40 to 80 years who have early Parkinson's Disease (PD) with increased activity in the LRRK2 pathway, identified through a genetic test. This Phase 2 trial aims to evaluate how well NEU-411 works and its safety in this specific group compared to a placebo. The study involves participants with clinically established or probable PD and focuses on those with LRRK2-driven PD. Participants will be randomly assigned to receive either NEU-411, a brain-penetrant oral drug that inhibits LRRK2 activity, or a matching placebo once daily for 52 weeks. After completing this treatment phase, participants will have a safety follow-up visit within two weeks. Those eligible may join an open-label extension to receive NEU-411 for an additional 26 weeks. During the study, researchers will monitor changes in a digital biomarker score using a Parkinson's Disease app from enrollment through 52 weeks, along with recording any treatment-emergent or serious adverse events until the study ends at 54 weeks. Assessments include genetic testing, clinical evaluations of PD status, safety monitoring, and tracking of side effects to understand the drug's effects and tolerability over time.

Researchers are evaluating the safety and effects of fosmanogepix, a study medicine, for treating candidemia and invasive candidiasis, which are serious fungal infections caused by Candida species. This Phase 3 clinical trial compares fosmanogepix to the standard treatment of caspofungin followed by fluconazole, aiming to show that fosmanogepix is not worse than the standard therapy by a margin of 15%. The study includes adult patients diagnosed with these infections. Participants will receive either fosmanogepix or caspofungin as an intravenous infusion daily at the study clinic. After the initial infusion phase, patients may switch to oral tablets of fosmanogepix or fluconazole capsules, which can be taken at the clinic or at home if discharged. Treatment duration varies by individual, lasting up to six weeks depending on infection clearance and symptom improvement. A follow-up visit will take place six weeks after stopping treatment. During the study, patients will undergo multiple visits to monitor their health and treatment response. Researchers will assess outcomes such as the proportion of patients alive at 30 days and the overall treatment success at the end of study treatment, up to day 42. Safety will be closely monitored throughout the study and during follow-up, ensuring comprehensive evaluation of the treatments over the entire participation period.

Researchers are evaluating the safety and effectiveness of fosmanogepix, given either intravenously or orally, for treating adults diagnosed with invasive mold infections. This Phase 3 study focuses on patients infected with molds such as Aspergillus species, Fusarium species, Lomentospora prolificans, Mucorales fungi, or other multidrug-resistant molds. The main goal is to compare the overall death rate at 42 days against a fixed threshold to assess treatment outcomes. Participants will be assigned to one of two groups: Cohort A includes patients receiving either the study drug fosmanogepix or the standard antifungal treatment based on institutional practice, while Cohort B includes patients receiving fosmanogepix as a salvage treatment after not responding to or not tolerating prior therapies. Fosmanogepix is administered through intravenous infusion or oral tablets. The study treatment period targets 84 days but can be extended up to 180 days depending on patient needs. Throughout the study, lasting up to approximately 8 months including follow-up, participants will undergo evaluations to monitor their response, safety, and overall health status. Researchers will track the all-cause mortality rate by Day 42 as the primary outcome. Safety and treatment effects will be assessed regularly during treatment and follow-up to ensure participant well-being and gather comprehensive data on fosmanogepix’s impact.

Researchers are conducting the X-TOLE3 Phase 3 clinical trial to evaluate the safety, tolerability, and effectiveness of XEN1101 as an additional treatment for adults with focal-onset seizures. The study focuses on measuring changes in seizure frequency when XEN1101 is added to existing antiseizure medications compared to placebo. Participants must have a confirmed diagnosis of focal epilepsy and have tried at least two antiseizure medications without achieving seizure freedom. About 360 participants will be randomly assigned in equal groups to receive either XEN1101 at 25 mg, 15 mg, or a placebo. The study includes up to 9.5 weeks of baseline observation to record seizure frequency, followed by 12 weeks of double-blind treatment where participants take the assigned capsules once daily with an evening meal. Those who complete this period may join a separate open-label extension to continue XEN1101 treatment, while others will enter an 8-week follow-up after treatment ends. During the study, participants will maintain accurate seizure diaries and continue stable doses of 1 to 3 antiseizure medications. Researchers will monitor seizure frequency changes from baseline through the 12-week treatment. Safety and tolerability will also be assessed throughout the trial. The total participation includes baseline, treatment, and follow-up periods to ensure thorough evaluation of the treatment's impact.

Researchers are studying the real-world effectiveness of pegcetacoplan in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This long-term, multicenter observational study aims to fill knowledge gaps about how pegcetacoplan works in routine medical practice. It also seeks to provide important information on red blood cell transfusions and healthcare resource use before and after starting pegcetacoplan treatment. Patients who have started pegcetacoplan treatment within the last 12 months or are prescribed it at enrollment will be followed for approximately 36 months. The study will collect both retrospective data from up to 12 months before treatment start and prospective data during treatment, with a total data collection period of up to about 48 months. After stopping pegcetacoplan, patients will remain in the study for 8 weeks to monitor for any adverse events. Participants will attend their usual medical visits, and data from these visits will be collected, including effectiveness measures, safety reports, patient- and clinician-reported outcomes, and healthcare use. The primary outcome includes changes in hemoglobin levels over 6 months from the start of pegcetacoplan treatment. The study plans to enroll about 200 patients across multiple countries, and data collection includes both retrospective and prospective periods, capturing comprehensive information on pegcetacoplan use in real-world settings.

Researchers are evaluating the long-term safety and effectiveness of etavopivat, a new oral medicine being developed to treat inherited blood disorders such as sickle cell disease and thalassemia. These disorders affect hemoglobin, the protein responsible for carrying oxygen in the body. This phase 3 study aims to monitor how well etavopivat works and its safety profile over an extended period. Participants will receive one of three forms of etavopivat (A, B, or C) as oral doses. The study is open-label and multicenter, involving adults, adolescents, and children who have previously completed treatment in an etavopivat parent study and continue to benefit clinically. The treatment period can last up to 264 weeks but may end earlier if etavopivat is approved in the participant's country. During the study, researchers will track the number of treatment-emergent adverse events and adverse reactions for each participant by indication and age group from baseline through the end of the study, which can last up to 316 weeks. Participants' safety and response to long-term treatment will be closely monitored throughout this period.

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.

Researchers are evaluating the effect of a triple therapy inhaler called BGF MDI containing budesonide, glycopyrronium, and formoterol fumarate compared with a dual therapy inhaler called GFF MDI containing glycopyrronium and formoterol fumarate in people with Chronic Obstructive Pulmonary Disease (COPD) who have a higher risk of heart and lung problems. This Phase III randomized, double-blind, parallel group study takes place at multiple centers and focuses on cardiopulmonary outcomes in these patients. Participants receive either the BGF MDI 320/14.4/9.6 micrograms twice daily or the GFF MDI 14.4/9.6 micrograms twice daily. The treatments are inhaled using metered dose inhalers. The study compares these two therapies over time to see how they affect the time until the first severe heart or lung event occurs. The study design ensures that neither participants nor researchers know which treatment is given to reduce bias. During the study, participants will have regular visits to the study site or virtual visits to complete assessments. Researchers will monitor lung function, symptoms, and blood tests, including blood eosinophil counts and COPD assessment test scores. The main outcome measured is the time to the first severe cardiac or COPD event, with follow-up lasting up to three years. Safety and adherence to treatment will also be closely observed throughout the study period.

Researchers are evaluating a Phase 2 study called PUMA-ALI-1201 to find the optimal dose of alisertib combined with endocrine therapy for adults with hormone receptor-positive, HER2-negative metastatic or recurrent breast cancer. Participants have experienced disease progression after at least two prior endocrine therapy treatments. The study also aims to assess the safety, effectiveness, pharmacokinetics of alisertib combined with endocrine therapy, and to identify biomarker-defined subgroups that may benefit most from this combination treatment. Participants will receive alisertib tablets orally twice daily on days 1-3, 8-10, and 15-17 of each 28-day cycle. They will also receive investigator-selected endocrine therapy according to approved dosing schedules, which may include daily oral tablets of anastrozole, letrozole, exemestane, tamoxifen, or intramuscular injections of fulvestrant on specified days. The combination treatment will be administered in repeated 28-day cycles. Throughout the study, researchers will monitor responses to treatment by measuring outcomes such as objective response rate, duration of response, disease control rate, progression-free survival, and overall survival for up to 48 months. Safety will be tracked by recording treatment-emergent adverse events from first dose through 28 days after the last dose. The study includes regular assessments to evaluate treatment effects, side effects, and participant well-being over the course of the trial.