Advancing Clinical Research

A clinical research study is a scientific investigation designed to answer important questions about an investigational drug, such as:


Is it safe?


Does it work?


Which dose works best?


What are the side effects?

This study is a Phase 2 study, which is evaluating the study drug’s safety and tolerability as well as whether the drug might work to treat PKU by lowering blood Phe. All study drugs must be tested in clinical research studies before they are approved and available to be prescribed by doctors. Clinical research studies are also reviewed and monitored by an independent Institutional Review Board (IRB), which makes sure that the rights of participants are protected and that there are no unnecessary risks involved in the study.

Choosing to take part in a clinical study is a big decision.

It is important for you to understand what the study is about, the potential risks, and what will be involved before you agree to participate. For more detailed information, you can reach out to a study site that is convenient for you.

Are you eligible?