Toledo

Researchers are evaluating the safety and effectiveness of elenestinib (BLU-263) combined with symptom-directed therapy (SDT) compared to placebo plus SDT in people with indolent systemic mastocytosis (ISM) whose symptoms are not well controlled by SDT alone. This Phase 2/3 randomized, double-blind, placebo-controlled study includes participants with ISM and smoldering systemic mastocytosis, and also involves groups for pharmacokinetic studies and participants who previously received a selective KIT inhibitor. The study is divided into multiple parts. Parts 1 and 2 enroll participants with ISM who will receive either elenestinib oral tablets or placebo alongside their symptom-directed therapy. Participants from Part 2 may continue into Part 3, which is an open-label extension where all receive elenestinib. Part K enrolls participants with ISM who have prior experience with selective KIT inhibitors. The study tracks treatment effects and safety over time. Participants will be monitored for up to 5 years, with assessments including the number of treatment-emergent adverse events, changes in symptom scores measured by the ISM-Symptom in Assessment Form, and overall safety monitoring. Evaluations occur at baseline, 13 weeks, 49 weeks, and throughout the long-term follow-up. The study also includes detailed tracking of symptom control and adverse events to evaluate the impact of treatment on participants' health and quality of life.

Researchers are evaluating the MammoWave, an innovative microwave mammogram device, to confirm its ability to detect breast lesions. This multicentric, single-arm, prospective study focuses on women with breast cancer or benign breast lesions, stratified by breast density. MammoWave uses very low power microwaves to create images without ionizing radiation, making it safe for use at any age and any condition, including pregnancy and specific illnesses. Participants will undergo the MammoWave exam, which takes about 10 minutes. The exam is performed with the patient lying face down in a comfortable prone position, with the breast placed in a special container cup within the device. The exam includes an acquisition phase where microwave data is collected followed by data processing through an integrated imaging algorithm, producing images and related parameters. The study includes two phases: a preliminary phase to optimize the imaging algorithm with volunteers having no lesions, and a second phase where patients with breast lesions will be examined and compared to standard clinical diagnosis. During the study, participants will have a short visit that includes a qualitative assessment of breast density. The MammoWave exam results will be compared with previous radiological studies and histological findings when available. Researchers will measure MammoWave's sensitivity in detecting breast lesions, tracking the number of true positive results. The study involves multiple sites in Italy, Germany, and Spain and plans to enroll up to 600 participants.

Researchers are evaluating the MammoWave device, which uses low-power microwaves instead of X-rays for breast cancer screening, in a large population of 10,000 women undergoing regular breast cancer screening programs. The study aims to confirm that MammoWave can achieve sensitivity greater than 75% and specificity greater than 90% in detecting breast cancer. This investigation is part of the MammoScreen project and involves multiple centers in Europe. Women participating in the study will first undergo a brief visit to check eligibility and review medical history. They will then have the MammoWave exam on both breasts, which includes an 8-minute data acquisition phase while lying prone on the device's bed, followed by data processing using specialized microwave imaging algorithms. The device will generate microwave images and classification results indicating the presence or absence of suspicious breast lesions. Participants will continue with their conventional breast screening examinations, such as mammograms, which serve as the reference standard for comparison. The study will monitor MammoWave's sensitivity and specificity during the procedure. Women aged 45 to 74 with average breast cancer risk and no symptoms are eligible, and the study excludes those with breast prostheses, prior breast cancer, certain genetic risks, pregnancy, or breast sizes too large for the device. The overall participation involves coordinating MammoWave testing with routine screening appointments and consenting to study procedures.

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 ELVN-001, a new drug, in adults with chronic myeloid leukemia (CML), including those with or without a specific T315I mutation. This early phase 1a/1b study aims to find the best dose of ELVN-001 for future studies by assessing its safety, tolerability, and how the body processes the drug. The study also looks at changes in a key leukemia marker called BCR-ABL1 to gather initial evidence of the drug's effect on CML. Participants will receive ELVN-001 orally once or twice daily. The trial includes a dose escalation period to identify recommended doses for further research. This first-in-human study will monitor patients closely to understand the safety profile and pharmacokinetics of ELVN-001. The drug is being tested in patients who have relapsed, are resistant, or cannot tolerate other tyrosine kinase inhibitors (TKIs). Throughout the study, participants will be monitored for adverse events, dose-limiting toxicities, and any significant lab or heart test abnormalities up to 28 days in phase 1a and for up to 3 years in phase 1b. Researchers will assess safety, tolerability, and leukemia markers regularly. The total duration of monitoring allows for a thorough evaluation of ELVN-001's effects and safety in adults with chronic phase CML.

The target population for inclusion in this study is breast cancer patients recently diagnosed (from January 2016) with unresectable locally advanced or metastatic disease (either after a recurrence or as first diagnosis). No treatment regimen will be protocol specified. This is an observational study in which clinical decisions concerning the optimum management strategy for a particular patient are taken independently of and/or prior to, any decision by the physician to invite a patient to participate in the study. The treating physician will make all treatment decisions according to his/her regular clinical practice independent of this study. Patients enrolled on the study are free to withdraw their informed consent for the use and disclosure of health information at any time and when asked, patients are not obliged to provide a reason. Patients may request discontinuation from the study at any time. The date and the reason for withdrawal or discontinuation from the study must be recorded in the electronic case report form (eCRF). An attempt will be made to determine the date of discontinuation and final status (i. e. withdrawal of consent, loss to follow-up, death) of any patient who discontinues from the study. However, the treating clinician is encouraged to follow the patient as long as possible, until patient death or through study end. The Sponsor has the right to terminate the study at any time. The Sponsor will notify the investigator if the study is placed on hold or if the Sponsor decides to discontinue the study.

Researchers are evaluating the effectiveness and safety of a combination treatment called triple therapy, which includes bempedoic acid, ezetimibe, and either atorvastatin or rosuvastatin. This study focuses on patients with primary hypercholesterolemia or mixed dyslipidemia who are at high or very high cardiovascular risk. The goal is to understand how well this combination lowers LDL cholesterol (LDL-C) in a real-world clinical setting. The study observes patients who have already started triple therapy within the last four weeks. No drugs are administered as part of this study; instead, it monitors the ongoing treatment with bempedoic acid combined with ezetimibe and either rosuvastatin or atorvastatin. The study measures LDL-C changes from baseline to eight weeks after starting triple therapy and continues follow-up for one year to assess lipid goal achievement, adherence to therapy, treatment changes, laboratory value shifts, and occurrence of cardiovascular events. Participants will have their LDL-C levels and other lab values assessed at baseline, eight weeks, and one year after starting triple therapy. Researchers will collect data on adverse events, adherence to treatment, and cardiovascular outcomes such as heart attack, stroke, death from cardiovascular causes, and coronary procedures during the follow-up year. The study also tracks treatment pathways and changes over this period to better understand real-world use and effectiveness of this triple therapy approach.

Researchers are evaluating the effectiveness of camizestrant compared to standard endocrine therapy in patients with early breast cancer that is estrogen receptor positive (ER+) and human epidermal growth factor receptor 2 negative (HER2-). These patients have an intermediate or high risk of cancer recurrence and have already completed local treatments such as surgery and possibly chemotherapy, alongside at least 2 years and up to 5 years of standard adjuvant endocrine therapy. The study is a Phase III, open-label trial designed to assess outcomes over a long term. Participants will be randomly assigned to receive either camizestrant, an oral selective estrogen receptor degrader, or one of several standard endocrine therapies including tamoxifen, anastrozole, letrozole, or exemestane, administered according to local approved guidelines. The treatment duration for both groups is planned to last 60 months. Eligible patients may have previously used CDK4/6 inhibitors, and the study will specifically include those with intermediate or high risk of recurrence as determined by clinical and biological markers. During the study, participants will be monitored for up to 10 years from the last patient's randomization to evaluate invasive breast cancer-free survival. Additional outcomes include invasive disease-free survival, distant relapse-free survival, overall survival, safety, and clinical outcome assessments. The study involves ongoing assessments of health status, treatment effects, and safety to determine the long-term benefits and risks of camizestrant compared to standard therapies.

Researchers are evaluating the effects of TX000045 in patients with pulmonary hypertension caused by heart failure with preserved ejection fraction (PH-HFpEF). This Phase 2, double-blind, randomized, placebo-controlled proof-of-concept study aims to assess two dosing regimens of TX000045 over a 24-week treatment period to understand its impact on pulmonary vascular resistance and safety profile. Participants will be randomly assigned to one of three groups: a placebo group receiving subcutaneous injections every two weeks, a group receiving Dose A of TX000045 subcutaneously every two weeks, and a group receiving Dose B of TX000045 subcutaneously every four weeks alternating with placebo every two weeks. The treatment period lasts for 24 weeks. Throughout the study, participants will undergo assessments including pulmonary vascular resistance measurements, physical examinations, laboratory tests, and monitoring for adverse events from baseline up to 30 weeks after the first dose. Safety evaluations focus on treatment-related side effects and changes in lab values. The study plans to enroll about 180 participants between 18 and 83 years old with specific heart and lung function criteria.

Parkinson's disease (PD) is a neurological condition that affects the brain, causing symptoms such as tremors, stiffness, and slow movement. This research aims to evaluate changes in sleep disturbances in adults with advanced Parkinson's disease who are receiving subcutaneous Foslevodopa/Foscarbidopa treatment. The study focuses on participants diagnosed with levodopa-responsive idiopathic Parkinson's disease experiencing severe motor fluctuations and hyperkinesia or dyskinesia. Participants will be treated with Foslevodopa/Foscarbidopa via subcutaneous infusion as prescribed by their doctors under routine clinical practice. Approximately 103 adults will be enrolled from around 20 sites across Spain. The treatment will follow the approved summary of product characteristics, and participants will be observed for up to approximately 12 weeks. During the study, participants will attend regular hospital or clinic visits according to their usual care schedule. Researchers will assess changes in Parkinson's disease-related sleep disturbances using the Parkinson's Disease Sleep Scale - 2 (PDSS-2). Cognitive function, treatment adherence, and any contraindications will also be monitored to ensure participant safety and study compliance throughout the observation period.