What Is a Clinical Research Study?

Clinical research studies, also called clinical trials, look at an investigational medication or medical device to see if it is safe, how it works in the body, and if it works to treat a specific disease. Clinical research studies are conducted by doctors who are responsible for the study participants’ study-related care.

In most countries, the regulatory health authority, such as the Food and Drug Administration (FDA) in the United States, requires that several phases of clinical research be performed to better understand the safety and effect of investigational medications and certain medical devices.

Clinical research studies must be approved by an institutional review board (IRB) or ethics committee (EC). An IRB/EC is a group that is responsible for helping to protect the rights and welfare of study participants. In addition, every study participant is monitored with study-related medical tests and exams before, during, and sometimes even after the study.

Can I Withdraw from the Study?

Participation in any clinical research study is completely voluntary, and you or your child may choose to withdraw from the study at any time for any reason. If you or your child would like to withdraw from the study, discuss this with the study doctor, who will provide information about how to do this safely.

What Should I Expect?

Before participants can take part in the Kayak Study, they will first need to attend the screening visit(s) for initial tests and assessments to see if they are eligible to participate. After all necessary tests and assessments have been completed, and if they are eligible to participate, they may enter as part of the Kayak Study and receive an investigational medication or placebo.

How Long Will Study Treatment Last?

Total treatment will last up to 18 weeks. In the first 6 weeks, participants will receive increasing doses of the study drug (titration period) based on weight, followed by 10 weeks of treatment at their final tolerated dose (maintenance period) and up to 2 weeks of treatment with decreasing doses (taper period) if the participant does not enroll in the separate extension study. Following completion of the Kayak Study, eligible participants, including those who received placebo, may have the option to enroll into an extension study to receive NBI-921352 for about 2 years.

Learn more about the Kayak Study.