Pembroke Pines

Researchers are studying adults with confirmed Primary Biliary Cholangitis (PBC) and cirrhosis, a scarring of the liver caused by damage to bile ducts. PBC is a slowly progressing disease that causes bile acid buildup and further liver damage, which can lead to cirrhosis. This study aims to evaluate if elafibranor, a daily medication, can prevent worsening clinical outcomes such as the need for liver transplant or death, compared to a placebo. It also looks at the safety of long-term elafibranor use and its effect on symptoms like itching and tiredness. Participants will take either an 80 mg tablet of elafibranor or a matching placebo once daily for up to 3.5 years in a double-blind setup, meaning neither the participants nor researchers know who receives which treatment. This long-term treatment period is designed to monitor the drug's impact over time. The study includes two groups: one receiving elafibranor and the other receiving placebo, with treatment lasting up to approximately 42 months. During the study, participants will be regularly assessed from the start until 4 weeks after treatment ends, with a maximum involvement of 3.5 years. Researchers will measure event-free survival, tracking if participants avoid clinical events indicating disease worsening. Safety monitoring will include tracking side effects and overall health, while symptom impact will be evaluated. Participants will provide informed consent and follow the study protocol throughout this extended observation period.

Researchers are evaluating the safety and effectiveness of combining ruxolitinib, steroids, and lenalidomide in patients with relapsed or refractory multiple myeloma (MM) who show disease progression. Multiple myeloma is a cancer of plasma cells in the bone marrow, and despite advances in treatment, it remains incurable. This phase 1, open-label, multicenter study aims to explore new therapeutic options by targeting the JAK/STAT pathway involved in MM cell growth and survival. Participants will receive oral ruxolitinib daily on days 1 through 28, lenalidomide on days 1 through 21, and methylprednisolone daily on days 1 through 28 of each treatment cycle. The combination therapy is being studied to determine the maximum tolerated dose of ruxolitinib when used with steroids and lenalidomide. The study also monitors treatment-emergent adverse events over a period of up to 54 months. Throughout the study, participants will undergo various assessments including laboratory tests to monitor blood counts, liver and kidney function, and disease markers. Researchers will evaluate safety, tolerability, and efficacy by tracking adverse events and disease progression. Participants must be able to follow the study schedule and provide informed consent. The study also involves registration in the REVLIMID REMS program for safety monitoring related to lenalidomide use.

Researchers are investigating the effects of two different doses of Glycopyrronium (GP) metered dose inhaler (MDI) compared to a placebo MDI when added to background treatment with Budesonide and Formoterol Fumarate (BFF) MDI. This study focuses on children aged 4 to less than 12 years who have asthma. The goal is to assess how these treatments affect lung function in this pediatric population during a Phase II clinical trial. The study is designed as a multi-center, randomized, double-blind, 3-period, 6-sequence crossover trial. It begins with a 3-week run-in period, followed by three separate 3-week treatment periods during which participants receive one of the three treatments: BFF MDI plus GP MDI Dose A, BFF MDI plus GP MDI Dose B, or BFF MDI plus placebo MDI. All inhalers are taken twice daily via oral inhalation. After completing the treatment periods, participants attend a safety follow-up visit 12 to 16 days after their last dose. Participants will undergo regular assessments including lung function tests to measure Forced Expiratory Volume in one second (FEV1) one hour after dosing at the end of treatment. Researchers will monitor safety through clinical exams and follow-up visits. The total participation duration includes the run-in, treatment periods, and safety follow-up, providing a comprehensive evaluation of the treatments' effects on asthma control in children.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study to see if it can reduce the risk of major adverse cardiovascular events (MACE-PLUS) in adults with established atherosclerotic cardiovascular disease (ASCVD) or those at high risk for a first ASCVD event. The study compares AZD0780 to a placebo and monitors participants from randomization until the primary analysis censoring date, followed by a final study closure visit. Participants will be randomly assigned to receive either oral AZD0780 or an oral placebo once daily. The treatment period lasts until the primary analysis censoring date, after which a study closure visit will occur. The study is event-driven and designed to assess the time to the first major cardiovascular event during treatment. During the study, participants will be closely monitored with various assessments to evaluate cardiovascular outcomes and safety over approximately 54 months. Researchers will track the time to first event of any component of MACE-PLUS and collect data to assess the effect of AZD0780 compared to placebo. The study includes regular visits and evaluations to ensure participant safety and adherence to treatment.

Researchers are evaluating insulin icodec, a once-weekly insulin injection, compared to insulin glargine, a once-daily injection, in adults with type 1 diabetes. The study aims to see how well weekly insulin icodec controls blood sugar levels compared to daily insulin glargine when both are combined with insulin aspart. This phase 3 study will last about 26 weeks, or roughly 8.5 months. Participants will receive either insulin icodec or insulin glargine, both given as subcutaneous injections. All participants will also use insulin aspart as a subcutaneous injection. The study compares these two insulin regimens to assess their effects on blood sugar control over the 26-week period. During the study, researchers will monitor changes in glycosylated hemoglobin (HbA1c) from the start of the study to week 26. Participants will follow the study protocol including self-measured plasma glucose profiles. Safety and efficacy will be evaluated throughout the treatment period to understand the impact of the insulin regimens on blood sugar control and participant health.

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 evaluating efruxifermin (EFX) in adults aged 18 to 80 who have compensated cirrhosis caused by nonalcoholic steatohepatitis (NASH) or metabolic dysfunction-associated steatohepatitis (MASH). This Phase 3, randomized, double-blind, placebo-controlled study aims to assess the safety and effectiveness of EFX in improving liver health and delaying disease progression in this population. The study focuses on subjects with advanced liver fibrosis (stage 4) but without liver decompensation. Participants are randomly assigned to receive either efruxifermin or a placebo, both administered by subcutaneous injection. The study includes two cohorts: Cohort 1 requires biopsy confirmation of liver fibrosis and specific metabolic features, while Cohort 2 allows biopsy or non-invasive diagnosis. Treatment and observation continue over an extended period to evaluate changes in liver fibrosis and clinical events. During the study, researchers will monitor the time until significant clinical events such as disease progression or liver decompensation occur, with a follow-up of up to five years. For Cohort 1, the proportion of participants showing improvement in fibrosis without worsening steatohepatitis will be assessed at 96 weeks. Participants will undergo regular evaluations including clinical assessments and laboratory tests to track liver function and safety throughout the study period.

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 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.

The primary purpose of the study is to assess how well amivantamab in combination with lazertinib or in combination with chemotherapy works (antitumor activity) in participants with epidermal growth factor receptor mutated (EGFRm) non-small cell lung cancer (NSCLC; that is one of the major types of lung cancer).