Clinical Trials

A clinical trial is a biomedical or health-related research study conducted in humans to answer specific questions about potential new treatments for disease. In most cases, the drugs being investigated in clinical trials have not yet been approved for marketing and sale by regulatory agencies (such as the U.S. Food and Drug Administration, or FDA), and so the benefits and risks of taking the treatment may not be known. Clinical trials are carried out to determine whether new treatments are safe and effective for patients.

Prior to regulatory approval, the human clinical testing of investigational treatments or drugs is generally done in three phases, with each successive phase designed to increase the understanding of the drug's effect on the body in terms of safety as well as its impact on disease. Until marketing approval is obtained, a drug will be continually evaluated through clinical trials to identify the most optimal dose and dosing regimen of the drug. The goal of this process is to be able to administer the drug safely and demonstrate clinical benefit for the intended patient population, and thus ultimately find novel and better treatment options for patients.

Alnylam's Phase IIb trial with ALN-RSV01 in lung transplant patients with respiratory syncytial virus (RSV) is now complete. For more information about the trial, click here

Alnylam's Phase I trial with ALN-VSP in patients with cancer involving the liver is now complete. For more information about the trial, click here

To learn more about Alnylam's Phase I trial with ALN-TTR01 in patients with transthyretin-mediated amyloidosis (ATTR), click here

To learn more about Alnylam's Phase I trial with ALN-PCS02 in healthy volunteers with elevated LDL-cholesterol, click here