Alnylam Pharmaceuticals

This study tests an experimental drug called zilebesiran in 11,000 adults whose high blood pressure is not well controlled despite taking at least two standard medications. The goal is to see if adding zilebesiran can lower the risk of major heart problems like heart attack, stro…

This study tests a new drug called ALN-6400 for hereditary hemorrhagic telangiectasia (HHT), a condition that causes abnormal blood vessels and frequent nosebleeds. The trial has two parts: first, healthy volunteers get a single dose to check safety; then, HHT patients get multip…

This early-stage study tests a new drug called ALN-BCAT, given alone or with an immunotherapy (pembrolizumab), in people with advanced liver cancer that has spread. The main goals are to check safety, find the best dose, and see if the drug shrinks tumors. About 158 adults whose …

This study tests an experimental drug called nucresiran in people with a rare disease that causes abnormal protein buildup in the heart (ATTR amyloidosis with cardiomyopathy). The goal is to see if the drug can lower the risk of death and serious heart events compared to a placeb…

This early-stage study tests a new drug called ALN-5288 in 50 adults with mild Alzheimer's disease or related memory problems. The drug is given via a spinal injection and aims to reduce a harmful protein called tau in the brain. The main goals are to check safety and how the dru…

This study tests a new medicine called ALN-AGT01 RVR in adults with mild to moderate high blood pressure who have already been treated with another drug, zilebesiran. The goal is to see if it safely helps lower blood pressure. About 93 participants will receive either the study d…

This study tests an experimental drug called ALN-2232, alone or together with an approved weight-loss medication (tirzepatide), in adults with obesity. The goal is to see if the combination is safe and helps people lose more weight. About 156 participants with a BMI between 30 an…

This early-stage study tests a new drug called ALN-HTT02 in adults with Huntington's disease. The main goal is to check if the drug is safe and how it moves through the body. Researchers will also measure if it lowers the level of the harmful protein that causes the disease. The …

This study tests a new drug called nucresiran in people with a rare inherited disease that damages nerves (hATTR-PN). The goal is to see if it can improve nerve function and quality of life compared to an existing treatment. About 125 adults will take part in this global Phase 3 …

This study tests a new medicine called ALN-4324 in two groups: first in healthy overweight volunteers to check safety, then in overweight adults with type 2 diabetes to see if it helps control blood sugar. The study is early-stage and focuses on safety and how the drug works in t…

This early-stage study is testing a new drug called ALN-APP for people with early-onset Alzheimer's disease. The drug is given as a shot into the spine to see if it is safe and how it affects the body. About 60 adults with mild memory problems or mild dementia will take part. The…

This study tests whether a single dose of the experimental drug ALN-4324 can improve how the body responds to insulin in people with type 2 diabetes. About 40 adults on stable metformin therapy will receive either the drug or a placebo. The main goal is to measure changes in insu…

This study tests an experimental drug called ALN-APP in 200 adults with cerebral amyloid angiopathy (CAA), a condition that causes bleeding in the brain. The drug is given via spinal injection and aims to slow disease progression by reducing new brain bleeds. Participants will be…

This study follows 2,000 adults with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) who are starting vutrisiran or another approved therapy. Researchers will observe how the disease progresses, how patients feel, and what treatments they receive in routine medical care. …

This study monitors women who were exposed to the drug patisiran during pregnancy to see how their pregnancies and babies turn out. It aims to identify any birth defects or complications. About 10 participants will be followed, and no new treatments are being tested.

This study is a global registry that will follow about 150 people with acute hepatic porphyria (AHP) over time. Researchers will collect information on how the disease progresses, how it is managed in everyday medical practice, and the safety and effectiveness of approved treatme…

This study follows 1,500 people with ATTR amyloidosis, a rare disease where abnormal proteins build up in the body. Researchers will track how the disease progresses and how well current treatments work in everyday medical practice. The goal is to better understand the condition …

This study looks at a protein called NfL in the blood of people who carry a gene linked to a rare disease that damages nerves (hATTR amyloidosis). Researchers want to see if NfL levels can help detect when the disease starts, how it progresses, and how well treatments work. About…

This early-stage study is testing the safety of a new drug called ALN-4915 in 44 healthy adults. Participants receive either the drug or a placebo, and researchers monitor for side effects and how the drug moves through the body. This study does not aim to treat any disease, but …

This early-stage study tests the safety and tolerability of a new drug called ALN-4285 in 76 healthy adults. Participants receive a single injection of either the drug or a placebo, and researchers monitor for side effects and how the drug moves through the body. The goal is to g…