Mayaguez

Researchers are evaluating the safety and tolerability of DB-1303BNT323 in adults with advanced or metastatic solid tumors that express HER2. This Phase 12a trial focuses on patients with tumors that are advanced, unresectable, recurrent, or metastatic and have limited or no standard treatment options. The study aims to identify the best dose and explore early signs of effectiveness in a variety of HER2-expressing cancers. The trial has two parts an initial dose-escalation phase using an accelerated titration followed by a classic 33 design to find the maximum tolerated dose MTD or recommended Phase 2 dose RP2D, and a dose-expansion phase to further assess safety, tolerability, and potential effects at the established dose. Participants receive DB-1303BNT323 by intravenous infusion once every three weeks Q3W at various dose levels. Some groups are randomized to receive different dose levels or combinations with other drugs like Pertuzumab, Ritonavir, or Itraconazole to study drug interactions and responses. During the study, participants will have regular assessments including monitoring for dose-limiting toxicities, adverse events, and serious adverse events using standard criteria up to about one year after treatment. Researchers will also evaluate tumor responses using RECIST 1.1 criteria and collect pharmacokinetic and pharmacodynamic data. Other evaluations include heart function tests, organ function, and overall health status. The study duration varies per participant, with follow-up visits extending up to one year post-treatment to monitor safety and treatment effects.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study. This trial focuses on patients with established atherosclerotic cardiovascular disease ASCVD or those at high risk for a first ASCVD event. The study aims to assess how AZD0780 compares to placebo in reducing the risk of major adverse cardiovascular events, also known as MACE-PLUS, over the course of the trial. Participants are randomly assigned to receive either oral AZD0780 once daily or a matching placebo once daily. The study continues until a primary analysis censoring date, which may be up to approximately 54 months from randomization. After this, a study closure visit will be conducted as the final visit for each participant. During the study, participants will be regularly monitored for cardiovascular events including heart attacks, strokes, urgent coronary revascularizations, and other related outcomes. Researchers will track the time to first occurrence of these events as the primary outcome. Safety and other secondary outcomes like all-cause mortality will also be assessed. The total participation time can last up to about 54 months, with ongoing evaluations throughout this period.

Researchers are evaluating efruxifermin EFX in a phase 3, randomized, double-blind, placebo-controlled study involving adults with compensated cirrhosis caused by NASH Nonalcoholic Steatohepatitis or MASH Metabolic Dysfunction-Associated Steatohepatitis. This study aims to assess the safety and effectiveness of EFX in preventing significant clinical events such as disease progression and liver decompensation over a period of up to 5 years. Participants are randomly assigned to receive either efruxifermin 50 mg or a placebo, both given by subcutaneous injection. The study includes two cohorts one with biopsy-proven compensated cirrhosis and specific metabolic scores, and another with biopsy-proven or non-invasive diagnosis of compensated cirrhosis. The study treatment and monitoring extend up to 5 years, with evaluations at 96 weeks and long-term follow-up to track liver fibrosis, markers of liver injury, insulin sensitivity, glycemic control, body weight, and safety outcomes. During the trial, participants undergo regular assessments including laboratory tests, ECGs, ultrasounds, and vital sign monitoring. Researchers will measure changes in liver fibrosis, steatohepatitis resolution, and metabolic markers throughout the study. Safety and tolerability are closely tracked by documenting adverse events and exposure duration. The study duration allows for long-term observation of treatment effects and disease progression, with participant involvement lasting up to 5 years.

Researchers are evaluating efruxifermin EFX in adults with non-cirrhotic nonalcoholic steatohepatitis NASH or metabolic dysfunction-associated steatohepatitis MASH who have liver fibrosis stage 2 or 3. This Phase 3, multi-center, randomized, double-blind, placebo-controlled study aims to assess the safety and efficacy of EFX compared with placebo. The trial includes about 1,650 participants divided into two cohorts based on liver biopsy characteristics and fibrosis stage. Participants will be randomly assigned to one of three groups EFX 28 mg, EFX 50 mg, or placebo, each given as a weekly subcutaneous injection. Cohort 1 will be evaluated over 52 weeks for histologic efficacy endpoints, while Cohort 2 will have assessments over 96 weeks. After these periods, participants may continue long-term treatment and clinical follow-up for up to approximately 240 weeks total. A follow-up visit will occur about 30 days after the last dose. During the study, participants will undergo liver biopsies, blood tests, and non-invasive assessments such as FibroScan and Enhanced Liver Fibrosis ELF score to monitor liver health and fibrosis. Researchers will track liver-related clinical outcomes, including liver events and survival, as well as safety and tolerability of the treatment. Participants who stop the study drug may still continue with scheduled assessments to support long-term safety and efficacy evaluations.

Researchers are evaluating the efficacy and safety of two different dose regimens of pegozafermin compared to a placebo in adults with metabolic dysfunction-associated steatohepatitis MASH who have liver fibrosis stage F2 or F3. This Phase 3 study aims to better understand how pegozafermin may impact liver fibrosis and steatohepatitis in this population. Participants will receive subcutaneous injections of either one of two pegozafermin regimens or a matched placebo. These treatments are given in parallel groups, and participants are randomly assigned to one of the study groups. The study compares the effects of pegozafermin on liver fibrosis and steatohepatitis over a treatment period that includes evaluations up to 52 weeks and monitoring for disease progression up to 5 years. During the study, participants will be monitored through biopsies and blood tests to assess liver fibrosis improvement, resolution of steatohepatitis, changes in liver enzyme levels, and enhanced liver fibrosis scores. Safety and disease progression are also tracked throughout the study period. The total participation duration includes treatment and long-term observation to evaluate outcomes and any potential changes in liver health.

Researchers are evaluating BMS-986365 compared to the investigators choice of therapy in men with Metastatic Castration-resistant Prostate Cancer. This phase 3, randomized trial aims to assess how well BMS-986365 works and how safe it is, focusing on radiographic progression-free survival. The study includes participants who have previously been treated with androgen receptor pathway inhibitors and have metastatic prostate cancer confirmed by imaging. Participants are randomized into groups receiving either one of two dose levels of BMS-986365 or an active comparator treatment chosen by the investigator, which includes either Docetaxel plus PrednisonePrednisolone or Enzalutamide or Abiraterone plus PrednisonePrednisolone. The study has two parts Part 1 compares the different doses and comparator arms, while Part 2 focuses on the selected BMS-986365 dose versus the investigators choice. Dosing schedules are specified but not detailed here. During the study, participants undergo regular assessments including imaging scans to evaluate cancer progression, pain and symptom questionnaires, blood tests, electrocardiograms, and monitoring for adverse events. Outcomes measured include progression-free survival, overall survival, response rates, pain progression, and quality of life changes. The study may last up to 4 years, with ongoing safety and efficacy evaluations throughout this time.

Researchers are evaluating the efficacy and safety of pegozafermin in adults with compensated cirrhosis caused by metabolic dysfunction-associated steatohepatitis MASH, previously known as nonalcoholic steatohepatitis NASH. This study focuses on participants with biopsy-confirmed advanced liver fibrosis stage F4 due to MASH. The research aims to understand how pegozafermin affects liver health over time compared to a placebo. Participants will receive either pegozafermin or a matched placebo through subcutaneous injections. The study follows a randomized, parallel design with quadruple masking to ensure unbiased results. The treatment period extends up to 24 months, with additional long-term follow-up lasting up to five years to assess disease progression and liver fibrosis regression. During the study, participants will undergo various assessments including measurements of liver fibrosis, disease progression through clinical events, and liver function tests such as alanine aminotransferase ALT levels. Tools like Enhanced Liver Fibrosis ELF score and FibroScan Vibration-controlled Transient Elastography VCTE will be used to monitor liver condition up to 60 months. Safety and efficacy will be closely monitored throughout the study period, which may last up to seven years in total.

Researchers are evaluating the efficacy, safety, and tolerability of adding ponsegromab to systemic chemotherapy compared to chemotherapy plus placebo for adults with metastatic pancreatic ductal adenocarcinoma mPDAC who have cachexia, a condition causing significant weight loss and fatigue. This Phase 2b3 randomized, double-blind, multinational study focuses on first-line treatment for this advanced cancer and associated cachexia. Participants will receive standard first-line chemotherapy regimens, either nab-paclitaxel plus gemcitabine or FOLFIRINOX, combined with either ponsegromab at one of two doses or a matching placebo. Study intervention is given subcutaneously every four weeks starting on the same day as the chemotherapy cycle and prior to chemotherapy administration. After Phase 2b, one ponsegromab dose will be selected for Phase 3, and participants will either continue or switch to that dose while remaining blinded. An optional open-label extension allows participants to receive ponsegromab for up to 12 months after the double-blind phase. During the study, participants will have tumor assessments approximately every 6 to 8 weeks by independent radiologists. Researchers will measure changes in body weight, anorexia symptoms, physical activity, muscle and fat tissue quality, overall survival, and treatment safety through laboratory tests, adverse event monitoring, and patient questionnaires. The study duration extends through Phase 3 with ongoing monitoring until key survival events occur, with an additional optional sub-study assessing caregiver quality of life.

This trial focuses on adults with primary IgA nephropathy IgAN who have already completed 9 months of treatment with TARPEYO4 delayed-release budesonide capsules at 16 mg once daily in regular clinical practice. The study aims to determine if continuing TARPEYO4 treatment provides further benefits in reducing proteinuria and protecting kidney function over a longer period. This is a phase 4, open-label study sponsored by Calliditas Therapeutics AB. Participants will undergo an extended treatment regimen lasting about 15 months, starting with 6 months of TARPEYO4 16 mg once daily, followed by 9 months of TARPEYO4 8 mg once daily. After this, there will be a 3-month follow-up period, including a 2-week tapering phase with TARPEYO4 4 mg once daily. The goal is to maintain or improve treatment effects while reducing dosage gradually. During the study, participants will have urine tests, blood samples, and physical exams at regular intervals to monitor kidney function and protein levels. Researchers will measure the urine protein to creatinine ratio UPCR and estimated glomerular filtration rate eGFR at various points, assessing safety by tracking adverse events over approximately 19 months. The study includes thorough follow-up to evaluate the ongoing impact of extended TARPEYO4 use.

Researchers are evaluating palazestrant OP-1250 compared to standard endocrine therapies for adults with ER-positive, HER2-negative advanced or metastatic breast cancer that has progressed after endocrine therapy combined with a CDK46 inhibitor. This international phase 3 trial aims to assess the safety and effectiveness of palazestrant versus fulvestrant or aromatase inhibitors such as anastrozole, letrozole, or exemestane. Participants are randomly assigned to receive either palazestrant daily on a 28-day cycle at doses of 90 mg or 120 mg during the dose-selection phase, or the standard-of-care endocrine therapy including fulvestrant administered on specific days or one of the aromatase inhibitors given daily on similar cycles. The trial includes an initial dose-selection period with about 120 participants, followed by a larger randomized phase with approximately 390 participants receiving the selected dose of palazestrant or standard treatment. Throughout the study, participants will be monitored for adverse events, dose adjustments, and drug discontinuations up to 16 weeks. Researchers will measure progression-free survival for up to two years and overall survival for up to four years after randomization. Regular assessments will include clinical evaluations and safety monitoring to observe the effects and tolerability of the treatments during the trial.