Santa Maria

Researchers are evaluating the safety and tolerability of Efimosfermin Alfa in adults aged 18 to 75 years who have known or suspected metabolic dysfunction-associated steatohepatitis (MASH) with fibrosis at stage F2 or F3. This Phase 3, randomized, double-blind, placebo-controlled study focuses on participants with non-alcoholic fatty liver disease and metabolic syndrome components, aiming to better understand treatment effects in this population. Participants will receive either Efimosfermin Alfa injection or a placebo, with the study designed as a three-arm trial. The treatment will be administered according to the study protocol, though specific dosing details are not provided. The study will monitor participants over a period extending to at least 52 weeks, comparing the safety and tolerability of Efimosfermin Alfa against placebo. During the study, participants will be closely observed through clinical assessments including monitoring for treatment-emergent adverse events (TEAEs), laboratory tests to detect Grade 3 and Grade 4 abnormalities, and evaluation of any adverse events leading to discontinuation of treatment. These safety and tolerability measures will be recorded at Week 52, helping researchers assess the impact of Efimosfermin Alfa over time.

This research aims to evaluate the safety and effectiveness of efimosfermin alfa in improving liver fibrosis and resolving steatohepatitis in adults with metabolic dysfunction-associated steatohepatitis (MASH) confirmed by biopsy showing stage F2 or F3 fibrosis. The study compares efimosfermin alfa to a placebo and focuses on individuals with confirmed liver damage and metabolic syndrome features. Participants will receive either efimosfermin alfa or placebo, administered as a drug treatment. The study is designed as a phase 3, randomized, double-blind, placebo-controlled trial with three groups. Treatment effects will be assessed over 52 weeks, with a primary focus on liver fibrosis and steatohepatitis changes. The study includes long-term monitoring of liver-related clinical outcomes up to 48 months after randomization. During the study, participants will undergo liver biopsies confirmed by central pathology review, and researchers will monitor liver function and fibrosis improvement. Outcome measures include the proportion of participants showing fibrosis improvement without worsening steatohepatitis, resolution of steatohepatitis with stable fibrosis, and time to liver-related clinical events. Safety and efficacy will be closely evaluated throughout the treatment and follow-up periods.

Researchers are evaluating the safety, tolerability, and biomarker effects of VS-041 in adults aged 50 years and older who have Heart Failure with Preserved Ejection Fraction (HFpEF). This phase 1 trial focuses on participants diagnosed according to established criteria and classified as NYHA Functional Class II or III, with specific heart function and biomarker thresholds. The study aims to better understand how VS-041 works in this population by monitoring heart-related biomarkers and side effects over time. Participants will receive either a high dose or low dose of VS-041, or a matching placebo tablet, taken twice daily. The study treatment lasts for 28 days, during which researchers will track changes in specific blood biomarkers such as NordicPRO-C6, endotrophin, and NT-proBNP. These biomarkers help measure the heart condition and response to the treatment. The trial includes careful dosing and monitoring to assess safety and tolerability. During the study, participants will undergo regular assessments to check for any treatment-emergent adverse events and changes in biomarker levels from baseline up to day 28. The evaluations may include blood tests and clinical exams to monitor heart function and overall health. Participants are required to maintain stable doses of their usual heart failure medications and comply with trial procedures for the entire duration. The total participation period is focused on the 28-day treatment and observation window.

Researchers are investigating the safety and effectiveness of efruxifermin in people with non-cirrhotic nonalcoholic steatohepatitis (NASH) or metabolic dysfunction-associated steatohepatitis (MASH) who have moderate to advanced liver fibrosis (stage 2 or 3). This Phase 3 study is randomized, double-blind, and placebo-controlled, enrolling a total of 1650 participants in two groups to evaluate treatment outcomes. Participants will receive either efruxifermin or a placebo by subcutaneous injection. The study involves two cohorts, with Cohort 1 including patients who have biopsy-confirmed NASH or MASH and specific liver fibrosis and activity scores. The treatment period and detailed dosing schedules are not provided but the study compares the effects of the active drug against placebo. During the study, participants will be monitored for improvement in liver disease status, including resolution of NASH/MASH and at least a one-stage improvement in liver fibrosis after 52 weeks for Cohort 1. Long-term outcomes such as event-free survival will be observed over 240 weeks. Safety and efficacy assessments will be conducted throughout the study period, including evaluations of liver histology and metabolic health.

Researchers are evaluating the safety, tolerability, early effectiveness, and how the body processes efimosfermin in adults aged 18 to 75 who have metabolic dysfunction-associated steatohepatitis (MASH) with compensated cirrhosis confirmed by liver biopsy showing stage F4 fibrosis. This phase 2 study compares efimosfermin to a placebo to better understand its effects in this condition. Participants will receive either efimosfermin or a placebo through injections under the skin. The study is randomized, double-blinded, and placebo-controlled, meaning neither the participants nor the researchers know who receives the active drug or placebo. Treatment and follow-up will continue for up to 100 weeks to monitor safety and response. Throughout the study, researchers will track any treatment-emergent adverse events from the first day up to 100 weeks. Participants will be monitored regularly to assess their health and liver condition, ensuring their safety during the trial. The study aims to gather important information about how effective and safe efimosfermin is for people with MASH and compensated cirrhosis.

This research aims to evaluate the safety and effects of the study medicine PF-07328948 for adults with heart failure. It focuses on how this medicine works compared to a placebo in people who are already using standard heart failure treatments that include sodium-glucose cotransporter 2 (SGLT2) inhibitors. The trial is a Phase 2 study designed to better understand if PF-07328948 is safe and effective for managing heart failure symptoms and improving patients' health. Participants will be randomly assigned to receive either placebo tablets or one of three doses of PF-07328948 (low, medium, or high dose). All medications are taken once daily by mouth for 36 weeks. The treatment period is followed by ongoing study visits to monitor participants. The study involves 15 visits over about 48 weeks, with 10 visits at the study site and 5 visits conducted remotely by phone. During the study, researchers will assess participants at the start and after 36 weeks by measuring clinical events, changes in the six-minute walk test distance, and changes in heart failure symptoms using the Kansas City Cardiomyopathy Questionnaire. Safety and treatment effects will be closely monitored through these visits and assessments throughout the study period.

Researchers are evaluating the effectiveness of active surveillance and chemotherapy treatments in pediatric, adolescent, and adult patients with low risk and standard risk germ cell tumors. This phase III trial focuses on monitoring patients after tumor removal and comparing the outcomes of carboplatin-based versus cisplatin-based chemotherapy regimens. The study aims to maintain high overall survival rates for low risk patients and to compare event-free survival between the two chemotherapy options in standard risk patients. Additional objectives include assessing side effects such as hearing loss and neuropathy, and exploring tumor marker changes and other biological measures related to treatment outcomes. Patients with low risk stage I germ cell tumors undergo surgery followed by observation, with the option to transfer to standard risk treatment if the tumor recurs. Those with standard risk tumors are randomly assigned to one of four chemotherapy regimens combining bleomycin, etoposide, carboplatin, or cisplatin. Treatments are given intravenously on specific schedules every 21 days for up to 3 or 4 cycles, depending on the group. Throughout the trial, patients receive imaging scans, blood tests, tumor biopsies if needed, and pulmonary function tests to monitor treatment response and side effects. Participants are closely followed after treatment completion with regular visits every 2 months for the first year, then less frequently up to 10 years. Researchers collect data through imaging, blood samples, lung tests, and questionnaires to measure survival, disease recurrence, and side effects like hearing loss. The study also includes exploratory analyses of tumor markers and patient-reported outcomes to better understand treatment impacts and improve future care for germ cell tumor patients.

Researchers are evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of CK-4021586 in adults aged 40 to 85 years with symptomatic Heart Failure with Preserved Ejection Fraction (HFpEF). This Phase 2 dose-finding study focuses on participants who have NYHA functional class II or III heart failure and a left ventricular ejection fraction (LVEF) of 60% or higher. The study aims to gather important data on the drug's effects and safety profile in this specific heart condition. Participants will receive either oral CK-4021586 at doses of 150 mg, 300 mg, 450 mg, or 600 mg, or a matching placebo. Stable doses of background medications such as beta-blockers, ACE inhibitors, ARBs, or ARNIs are required before the study, as well as stable use of GLP-1 agonists if applicable. The study is randomized, double-blind, and placebo-controlled, ensuring rigorous comparison between the investigational drug and placebo groups. During the 12-week study period, participants will be monitored for early drug discontinuation, heart function changes indicated by LVEF falling below 40%, and the occurrence of adverse events. Evaluations include echocardiography, laboratory tests for NT-proBNP levels, and ongoing safety assessments. This careful monitoring helps researchers understand the treatment's safety and tolerability in people with HFpEF over the study duration.

Researchers are evaluating the safety and effectiveness of duvakitug in people with moderately to severely active Ulcerative Colitis (UC). This multinational, multicenter, randomized, double-blind, placebo-controlled Phase 3 study aims to see if duvakitug can help achieve clinical remission in this condition. The study targets participants aged 16 to 80 years with a confirmed diagnosis of active UC for at least 3 months who have not responded well or are intolerant to other treatments. Participants will receive either duvakitug or a placebo as a solution injected under the skin (subcutaneous injection). The study includes up to 35 weeks with multiple periods: a screening period, a 12-week induction phase (either open-label or randomized), a 12-week extended induction for those who do not respond initially, and a 45-day follow-up for those not continuing into the maintenance study. During these phases, participants may have up to 8 to 15 on-site visits depending on their sub-study group. Throughout the study, participants will be monitored closely with scheduled visits for assessments including clinical evaluations related to UC activity and response to treatment. The main outcome measured is the proportion of participants who achieve clinical remission by week 12. Safety and tolerability will also be tracked during and after the treatment period, with follow-up visits to ensure participant well-being.

Researchers are evaluating the addition of olaparib, a PARP inhibitor, as maintenance therapy following surgery and chemotherapy in patients with pancreatic cancer that has been surgically removed and who have a pathogenic mutation in BRCA1, BRCA2, or PALB2 genes. This phase II randomized, double-blind study aims to determine if olaparib can improve relapse-free survival compared to placebo in these patients, who have completed perioperative chemotherapy and have no evidence of recurrent disease. Participants are randomly assigned to receive either olaparib or a placebo orally twice daily in 28-day cycles for up to 12 cycles, as long as there is no disease progression or unacceptable side effects. Throughout the treatment period, patients undergo imaging tests such as CT scans or MRI and blood sample collections. After completing the treatment cycles, patients are followed up at 30 days, every 4 months for the first year, and then every 6 months for up to 10 years after randomization to monitor their health and disease status. During the study, researchers assess relapse-free survival by documenting any return of cancer or death from 22 to 44 months after randomization. They also collect blood samples and perform imaging tests to monitor the disease and evaluate treatment effects. Safety is carefully monitored, and patients must have recovered from previous treatments before starting the study. The study includes long-term follow-up to observe survival outcomes and any differences based on genetic mutations or prior chemotherapy regimens.