Santiago

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 investigating sovateltide, a new drug that targets ETB receptors, for treating acute cerebral ischemic stroke (ACIS), a condition caused by a blocked blood vessel in the brain leading to brain tissue damage. ACIS is the most common type of stroke and a serious emergency with limited treatment options. Current standard treatment with tissue plasminogen activator (t-PA) has a narrow time window and limited success in fully resolving stroke effects. Sovateltide has shown promise in animal studies and early human trials by promoting brain repair and improving neurological outcomes. The study compares sovateltide treatment alongside standard care to a placebo (normal saline) in patients who recently experienced ACIS. Participants will receive the study drug within 24 hours of stroke symptom onset. This phase III trial is randomized, double-blind, and placebo-controlled, conducted across multiple centers in the United States, Canada, the United Kingdom, and Europe. The trial builds on positive results from earlier phase II and III studies conducted in India. Participants will be followed for 90 days after treatment to assess recovery using scales that measure disability and neurological function. Researchers will monitor safety and effectiveness by evaluating participants' abilities and stroke outcomes, including the modified Rankin Scale score. The trial aims to provide further evidence on whether sovateltide can improve recovery and function after acute ischemic stroke when added to standard treatment.

Researchers are conducting a long-term observational study to monitor the safety and effectiveness of DTX401 in people with Glycogen Storage Disease Type Ia (GSDIa). This study follows participants for at least 10 years after they have received DTX401, either through earlier clinical studies or post-marketing treatment. The goal is to understand the lasting impact of this therapy over an extended period. Participants in this study will not receive any new investigational treatments as part of the monitoring program. Instead, the study collects data on those who have already been administered DTX401, whether a full or partial dose, in previous settings. The study includes two groups: those treated in parent clinical studies and those treated in a post-marketing context. During the study, researchers will track serious adverse events related to DTX401, including infusion-related reactions and the effects of any immunomodulatory therapies given alongside. Pregnancy incidence and outcomes among patients or their partners will also be monitored. Participants will be followed and assessed regularly over a 10-year period to gather comprehensive safety and effectiveness information.

Researchers are evaluating the effects of baxdrostat combined with dapagliflozin compared to dapagliflozin alone in adults aged 40 and older who have type 2 diabetes, established cardiovascular disease, a history of hypertension with systolic blood pressure of at least 130 mmHg at screening, and at least one additional risk factor for heart failure. This Phase III randomized, placebo-controlled, event-driven study aims to determine if the combination reduces the risk of heart failure events or cardiovascular death, with follow-up lasting up to 38 months. Participants who meet screening criteria but are not currently treated with SGLT2 inhibitors or have been treated for less than 4 weeks will enter a run-in period receiving dapagliflozin 10 mg once daily for 4 to 6 weeks before randomization. The study involves random assignment to either baxdrostat plus dapagliflozin or placebo plus dapagliflozin. Site visits occur at approximately 2, 4, 8, 16, and 34 weeks after randomization, then every 4 months. Participants discontinuing the blinded study drug may continue open-label dapagliflozin, with ongoing visits and data collection as per protocol. Participants will undergo an optional pre-screening period without site visits or consent to help identify eligibility, followed by up to 14 days of formal screening after informed consent. Researchers will monitor heart failure events and cardiovascular deaths as primary outcomes. Safety and adherence will be tracked throughout the study, including during any premature discontinuation of blinded treatment. The study will conclude when a predetermined number of secondary endpoint events have occurred, with continued follow-up as needed.