Mito

Researchers are evaluating AMG 732 in both healthy individuals and participants with moderate-to-severe active Thyroid Eye Disease (TED) in a Phase 1/2 clinical trial. The study aims to assess the safety, tolerability, pharmacokinetics, and efficacy of AMG 732 given by subcutaneous injection. Part A focuses on safety and tolerability after single doses in healthy participants, while Part B examines the efficacy of multiple doses in those with TED. Participants receive either AMG 732 or a placebo through subcutaneous injections. Part A involves single-dose administration to healthy adults aged 18 to 55 years, including a cohort of healthy Japanese participants. Part B enrolls adults aged 18 to 65 years with moderate-to-severe active TED, who receive multiple doses over approximately six months. The study design is randomized, double-masked, and placebo-controlled. During the trial, participants undergo various evaluations including safety monitoring for treatment-emergent adverse events up to 36 weeks, and measurement of proptosis (eye bulging) changes in the affected eye from baseline to the end of treatment. Assessments include clinical activity scores, laboratory tests, vital signs, ECGs, and physical exams. Researchers also track participants’ response to treatment and monitor for any side effects throughout the study duration.

Researchers are evaluating whether the medicine vicadrostat, combined with empagliflozin, helps adults with chronic heart failure (HF) who have a weakened heart pumping function, specifically a left ventricular ejection fraction (LVEF) below 40%. Eligible participants must have been diagnosed with chronic HF at least 3 months before joining. The study is a Phase III trial designed to compare the effects of vicadrostat plus empagliflozin against placebo plus empagliflozin in people with symptomatic chronic HF classified as New York Heart Association classes II to IV. Participants are randomly assigned to one of two groups. One group takes tablets containing vicadrostat and empagliflozin, while the other group takes placebo tablets that look like vicadrostat along with empagliflozin. Tablets are taken once daily for a period ranging from about 6 months up to about 3.5 years. Participants continue their usual heart failure treatments during the study. The study is double-blind, meaning neither the participants nor the study staff know who is receiving which treatment. During the study, participants regularly visit the study site or may have phone contacts for follow-up. They answer questions about their health and well-being. Doctors monitor and record any worsening of heart failure symptoms, hospital visits due to heart failure, or deaths. They also check participants' overall health and note any side effects. The main outcome measured is the time until a participant experiences cardiovascular death, hospitalization for heart failure, or an urgent heart failure visit, over up to 43 months of follow-up.

This study is open to adults aged 18 or above legal age with heart failure. People can join the study if they have heart failure symptoms and a left ventricular ejection fraction (LVEF) of 40% or more. The purpose of this study is to find out whether vicadrostat (BI 690517) in combination with empagliflozin helps people with heart failure. Participants are put into 2 groups by chance. Every participant has an equal chance of being in each group. The groups are: * Vicadrostat/empagliflozin group: participants take vicadrostat/empagliflozin as tablets once a day. * Placebo/empagliflozin group: participants take placebo/empagliflozin as tablets once a day. Participants can stay in the study as long as they benefit from treatment and can tolerate it. During this time, they visit their doctors regularly. The doctors regularly check participants' health and take note of any unwanted effects. The study staff may also contact the participants by phone. Participants also regularly answer questions about their well-being. The study does not have a fixed duration. It continues until there is enough data to see if the treatment is working.

Researchers are evaluating the effect of muvalaplin on reducing cardiovascular risk in adults with elevated lipoprotein(a) levels who either have atherosclerotic cardiovascular disease or are at risk for a heart attack or stroke. This Phase 3, randomized, double-blind, placebo-controlled study focuses on adults with high Lp(a) levels and prior or potential cardiovascular events. The study aims to assess the time to the first major adverse cardiovascular event over about 5.25 years. Participants will be randomly assigned to receive either muvalaplin or a placebo, both administered orally. The study includes individuals with Lp(a) levels of at least 175 nanomoles per liter who have had a prior cardiovascular event within 10 years or are at risk for a first event due to conditions such as coronary artery disease, carotid stenosis, peripheral artery disease, high coronary artery calcium score, reduced kidney function with diabetes, or other high-risk factors. The treatment period lasts through the study duration, with close monitoring. During the study, participants will be regularly evaluated to track the occurrence of major adverse cardiovascular events, including heart attacks and strokes. Safety assessments will monitor blood pressure, kidney function, and heart failure status among other health indicators. The primary outcome measures the time to the first major cardiovascular event from baseline up to the end of the study, which spans approximately 5.25 years.

Researchers are evaluating the effectiveness and safety of rimegepant compared to a placebo for preventing migraines in children and adolescents aged 6 to under 18 years who experience episodic migraine. This Phase 3 study focuses on participants who have had migraines for at least six months with a limited number of headache and migraine days per month. The study aims to understand how well rimegepant works to reduce the number of migraine days over a 12-week period. Participants will receive either rimegepant in doses of 75mg or 50mg (two 25mg orally disintegrating tablets) or a matching placebo. The treatment is administered over a double-blind 12-week phase where neither the participants nor the researchers know which treatment is given. This setup helps ensure unbiased results when comparing the preventive effects of rimegepant against placebo. Throughout the study, participants will be monitored for changes in their migraine frequency, specifically the average number of migraine days per month from the start to the end of the 12 weeks. Evaluations include headache and migraine tracking, as well as assessments of daily activity disruption. Safety and side effects will also be closely observed to understand the medication's impact on young patients.

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.

This research aims to test the safety and effectiveness of BHV-3000 (rimegepant) compared to a placebo for treating moderate to severe migraine attacks in children and adolescents aged 6 to under 18 years. The study focuses on pediatric migraine, including attacks with or without aura, lasting more than 3 hours and occurring 1 to 8 times per month over the previous two months. Participants must be able to clearly distinguish migraine from other headaches and weigh more than 40 kg. Participants will receive either BHV-3000 (rimegepant) at doses of 75 mg or 50 mg as an orally disintegrating tablet (ODT) or a matching placebo. The study is randomized, double-blind, and placebo-controlled, designed to evaluate the acute treatment effect. Some participants may continue stable migraine preventive medications, but use of CGRP antagonist drugs is not allowed. The treatment phase will monitor responses to the study drug during migraine attacks. During the study, participants will be assessed for migraine pain relief two hours after dosing, measuring how many experience complete freedom from pain. Blood samples will be taken, and participants will be monitored for safety and side effects. The study excludes those with certain headache types, significant psychiatric or neurological conditions, recent surgeries, or other serious medical issues that could affect participation or safety. Overall, the study evaluates both the effectiveness and safety of rimegepant in a pediatric population over the course of migraine attacks.

Researchers are evaluating a treatment for adults aged 50 and older who have macular neovascularization caused by neovascular age-related macular degeneration (nAMD). This Phase 3 trial compares a single intravitreal injection of 4D-150 with the active control treatment EYLEA (aflibercept). The study aims to assess changes in visual acuity over one year to better understand the effects of these treatments on this eye condition. Participants are randomly assigned to receive either a single dose of 4D-150 injected into the eye on Day 1 or ongoing EYLEA injections at scheduled visits. The study includes adults who have either never received anti-VEGF therapy or have had up to four prior anti-VEGF injections with documented improvement. The condition must be active and confirmed by specialized eye imaging tests before enrollment. Throughout the 52-week study, participants will have regular assessments including vision tests measured by the ETDRS letter score, eye imaging, and clinical evaluations to monitor treatment effects and safety. Researchers will track visual acuity changes from the start of treatment and watch for any adverse effects. The study's main goal is to measure the average change in best corrected visual acuity at one year.

This research aims to observe the safety and effectiveness of dabrafenib and trametinib in patients with BRAF V600E mutation-positive unresectable advanced or recurrent solid tumors, excluding colorectal cancer. It is a prospective, multicenter, single-arm, non-interventional observational study conducted through a central registration system using electronic data capture. The study includes both adult and pediatric patients, with long-term monitoring planned to collect comprehensive safety data. Patients already prescribed Tafinlar/Mekinist (dabrafenib and trametinib) before joining the study will be enrolled without treatment allocation or changes. The study targets 65 adult patients for effectiveness analysis and approximately 20 pediatric patients. Pediatric patients will be observed for up to 8 years after starting treatment to gather long-term information, while adult patients will be followed for one year post-treatment initiation. During participation, patient safety and treatment response will be monitored through reports of adverse events and overall response rates. Pediatric patients will have ongoing safety assessments related to skeletal and sexual development over the 8-year period. Adults will have their treatment response evaluated over one year. Data collection includes long-term follow-up regardless of treatment discontinuation, aiming to provide comprehensive post-marketing surveillance information on these medications.

Researchers are evaluating the effect of balcinrenone/dapagliflozin compared with dapagliflozin alone on cardiovascular death and heart failure events in patients with chronic heart failure and impaired kidney function who recently experienced a heart failure event. This is a Phase III, international, randomized, double-blind, parallel-group, active-controlled study involving approximately 700 sites in about 40 countries. Participants will be randomly assigned in a 1:1:1 ratio to receive one of three treatments once daily: a capsule of balcinrenone/dapagliflozin 15 mg/10 mg with a placebo tablet, a capsule of balcinrenone/dapagliflozin 40 mg/10 mg with a placebo tablet, or a dapagliflozin 10 mg tablet with a placebo capsule. The study is event-driven, with an estimated average duration of 22 months that includes a screening period, a 20-month blinded treatment phase, and a one-month follow-up on open-label dapagliflozin. During the study, participants will be monitored for the time to first occurrence of cardiovascular death, heart failure hospitalization, or heart failure events without hospitalization over approximately 38 months. Assessments include clinical evaluations, laboratory tests, and safety monitoring throughout the study and follow-up period to track treatment effects and patient outcomes.