• About Clinical Research

    Clinical research studies (also called clinical trials) are developed and done to learn more about diseases and conditions. Some studies are experimental, meaning they are designed to test investigational medications that may help treat particular diseases. Before any medication or medical advancement can be approved and made available to the general public, it has to go through several phases of experimental clinical research studies to make sure the treatment works safely. Other studies are observational, meaning they do not involve investigational medications and are only intended to observe participants and gather information to help researchers better understand particular diseases.


    This CTX study is observational, so there is no investigational study medication or treatment being tested or given to participants. This study is only being done to help researchers learn more about CTX.

    Why do people partic​ipate in studies?

    Study participation is completely voluntary. People participate in clinical research studies for a variety of reasons. Some volunteers may participate in studies because they want to learn more about their disease. Others volunteer to participate because they want to help researchers learn more about a disease so that it may help them and others in the future.

    Are clinical studies safe?

    Clinical research studies follow a specific set of standards and are closely regulated to ensure the rights, well-being, and safety of all participants. Safety precautions, regulations, and policies are put in place to protect people who participate in clinical research. These policies help ensure that these studies are conducted according to strict scientific and ethical principles. In addition, studies follow a written plan that is called a protocol. The protocol is designed to make sure all study procedures are conducted correctly and safely.


    The study and study-related documents must be reviewed and approved by an independent review board, which aims to protect the interests of study participants and ensure they are accurately informed about the research. This group is called an institutional review board (IRB) and is comprised of doctors, scientists, and members of the community.

    What if I have questions during the study?

    You can ask questions of the study doctor or staff at any point before, during, and after the study. Before agreeing to participate, please make sure that you understand the responsibilities of study participation. If you have any concerns about participating in the study, you should feel comfortable discussing them with a member of the study team at any time.

    Can I leave the study once it has started?

    Study participation is completely voluntary, and you can end your participation at any time, for any reason. If you think you would like to stop participating in the study, talk to the study doctor. If you decide you should leave the study early, the study doctor may ask that you return to the study site for a final visit, but there will be no negative impact on the care you receive.

    Can I see other doctors during the study?

    You can visit any doctor to meet your needs during the study. Please let the study doctor know that you will be seeing another doctor and if any medication is prescribed during the visit.