What You Should Know about Clinical Research Studies

What Is a Clinical Research Study?

Clinical research studies, also called clinical trials, look at a study drug 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 effectiveness of new study drugs 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 Leave the Study If I Change My Mind?

Participation in any clinical research study is completely voluntary, and participants may choose to leave the study at any time for any reason. You can leave the study at any time without giving a reason and without penalty or loss of benefits to which you are otherwise entitled. This would not affect your relationship with your doctor(s) or the level of care you receive in the future.

What Should I Expect?

Before you can take part in the CARAT or PERIDOT studies, you will be required to sign a study-specific informed consent form. Your first study visit will be the screening visit(s) for initial tests and assessments to see if you are eligible to participate. Depending on which study you prequalify for, a set of tests and procedures will be performed, and if you are eligible to participate, you may enter the applicable clinical research study. If you are participating in the CARAT study, you will either receive the study drug, venglustat, or standard-of-care treatment. If you are participating in the PERIDOT study, you will receive either venglustat or placebo (a substance that looks just like the study drug but contains no active ingredients).

How Long Will Study Participation Last?

Participation can last approximately up to three years in the CARAT study and approximately up to two years in the PERIDOT study.