Alnylam Pharmaceuticals

This large, late-stage study is testing whether an investigational drug called nucresiran can help people with a serious heart condition called ATTR amyloidosis cardiomyopathy. The main goal is to see if the drug reduces the risk of death and the need for urgent heart-related hos…

This early-stage study is testing a new drug called ALN-BCAT, both by itself and combined with an existing immunotherapy (pembrolizumab), for people with advanced liver cancer that has spread. The main goals are to find safe doses and see how well the body handles the treatment. …

This study is testing whether adding a new investigational drug called zilebesiran to standard blood pressure medications can lower the risk of serious heart problems like heart attack, stroke, or heart failure. It will involve about 11,000 adults with high blood pressure that is…

This early-stage study is testing a new drug called ALN-6400 to see if it can safely reduce severe nosebleeds and other bleeding problems in adults with hereditary hemorrhagic telangiectasia (HHT). The trial has two parts: first testing single doses in healthy volunteers, then te…

This study is testing a new drug called ALN-2232 to see if it is safe and helps people with obesity lose weight. Researchers will give it to participants by itself and also in combination with an existing weight-loss medication, tirzepatide. The main goals are to check for side e…

This is an early-stage study to test the safety and effects of a new drug called ALN-HTT02 for Huntington's disease. The drug is injected into the spinal fluid to try to lower the harmful protein that causes the disease. The study will involve 66 adults with mid-stage Huntington'…

This study is testing whether a medication called ALN-APP, given as an injection into the spinal fluid, can slow down the progression of cerebral amyloid angiopathy (CAA). CAA is a disease where a sticky protein builds up in the brain's blood vessels, making them fragile and pron…

This early-stage study is testing a new drug called ALN-4324. It aims to see if the drug is safe and can help control blood sugar in adults with type 2 diabetes who are also overweight or obese. The study will first test single doses in healthy volunteers, then test multiple dose…

This study is testing an investigational drug called nucresiran for people with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN), a rare disease that causes progressive nerve damage. The goal is to see if nucresiran can slow or stop the worsening of nerve funct…

This study is testing whether a single dose of an investigational drug called ALN-4324 can improve how well the body responds to insulin in people with type 2 diabetes. It will involve about 40 adult participants who are already taking a stable dose of metformin. Researchers will…

This is a first-in-human study to check if a new drug called ALN-5288 is safe for people with Alzheimer's disease. The drug is injected into the spinal fluid. Researchers will enroll 50 adults with mild to moderate Alzheimer's to monitor for side effects and see how the drug beha…

This early-stage study is testing the safety and effects of a new drug called ALN-APP for people with early-onset Alzheimer's disease. The drug is given as an injection into the spinal fluid. Researchers will monitor 60 participants to see how their bodies handle the drug and che…

This study aims to understand how ATTR amyloidosis affects people in real-world settings, not in controlled clinical trials. Researchers will follow 1,500 patients, including those with symptoms and those carrying the gene but without symptoms, to track disease progression and ho…

This study is creating a global registry to track 150 patients with acute hepatic porphyria (AHP), a rare genetic disorder. Researchers will follow patients over time to understand how the disease naturally progresses and how treatments work in real-world settings. The goal is to…

This is the first study in humans for the investigational drug ALN-4285. It aims to test the safety and how the body processes a single injection of the drug in 76 healthy adult volunteers. The study will start with low doses and gradually increase them to find a safe range for f…

This study aims to understand how patients with a serious heart condition called ATTR amyloidosis fare in everyday medical care, not just in clinical trials. Researchers will follow 2,000 patients for up to 5 years to see how treatments affect their quality of life, symptoms, and…

This study aims to see if a protein in the blood, called neurofilament light chain (NfL), can be a useful marker for a rare nerve disease called hATTR amyloidosis. It will measure NfL levels in 500 people who carry the gene for the disease but have no symptoms, and in those who a…

This study collects information about pregnancy outcomes for women with hereditary ATTR amyloidosis who took the medication patisiran. Researchers will track mothers and babies to understand any potential effects on pregnancy complications or infant health. The goal is to gather …