What are clinical studies (or “trials”)?

Clinical studies are medical research studies in which people participate as volunteers. Clinical research studies (sometimes called trials or protocols) are a means of developing new treatments and medications for diseases and conditions. There are strict rules for clinical trials. The process is regulated by the National Institutes of Health and the U.S. Food and Drug Administration, and all studies are monitored by a panel of experts to ensure they are conducted safely. The research studies at the Center for Rare Neurological Diseases involve promising new treatments that may directly benefit patients.

Why would I want to participate in a clinical trial?

You or your child may wish to participate in a clinical trial for a variety of reasons. Researchers can't discover new cures without your help. It can be a rewarding and satisfying experience as well as providing you an opportunity to contribute to the development of new and "cutting edge" medical treatment. You could be part of a process that is essential in the development of medications. All medications must go through this process to become FDA approved. The studies are performed to determine how well the medication works (efficacy) or how long it lasts (duration).

Especially in rare neurological disorders, you will gain unique access to experts in the disorder. The consulting physicians for the studies will provide you the opportunity to discuss your or your child’s disorder at length, and without charge.

People who volunteer for phase II and phase III trials can gain access to promising drugs long before these compounds are approved for the general public.

Study participants will receive close and comprehensive medical attention from highly skilled professionals who are readily available to answer any questions you have. In most studies, you will receive diagnostic testing, examinations and treatment without charge. This can help offset expenses that otherwise would make it difficult for you to receive care without insurance coverage.

Compensation for your time and travel may be provided.

Have these medications been tested before?

Most of our clinical trials are Phase II thru IV. This means that these medications have gone through both extensive testing in laboratories, and early human testing. Some of the clinical trials we conduct are for medications that have already been approved by the FDA. You will be informed of the background of any investigational drug or device before you enroll in a new study. In some studies there is also the chance that you may be assigned a placebo (tablet, treatment or device with no active medication). If you are participating in a study in which you might receive a placebo this will be explained to you before agreeing to start the study.

How long are the studies?

This depends on the study. The shortest studies can be as short as 2 weeks, and the longest can last several years. Most studies only last a matter of months.

What if I want to stop participating in the study?

You or your child’s participation is voluntary and you may choose not to participate at any time. There will be no penalty or loss of benefits to which you are otherwise entitled. If one of the consulting physicians is also your personal physician, they will not stop providing you medical care.

Are my rights as a person/volunteer protected?

YES. Aside from knowing that a study cannot begin without governmental approval, an independent group of professionals called an Institutional Review Board (IRB) also must approve the study before it can be initiated by the study physician. An IRB is a group of medical and non-medical professionals whose primary responsibility is to review the study to make sure it poses no undue risks to the study participants. Your physician will also be asked to provide the IRB with periodic reports while the study is being conducted. Additionally, your medical records are held in the strictest confidence. It is also important to know that you may terminate your participation at any time without jeopardizing your access to other medical treatments.

What is informed consent?

Before entering a clinical study, it is important that you as the patient and your child (if they are able) fully understand the study and what your involvement would mean. Our staff members will help by providing you with an informed consent statement, which has detailed information about the study, including the length of the study, the number of visits required, and medical procedures and medications included. It also provides expected outcomes, potential benefits, and possible risks.

We will review the informed consent statement with you and answer your questions. If you decide to participate after reviewing the statement and talking with staff and family members, you will need to sign the informed consent statement. If you are signing for your child, if your child is able to, your child may also have to “assent” to participate (agree even though they are not legally of age to sign the contract). Your signature indicates that you understand the study and agree to participate voluntarily. The informed consent tells patients that they have a right to leave the study at any time.

How do I know if I'm eligible to participate in a clinical trial?

Every study has a different purpose and specific scientific questions it is trying to answer. In order to do that, very specific criteria, or requirements, are developed to evaluate an individual's ability to participate. These criteria will determine if you are a good candidate for inclusion, or if there is any reason you should be excluded from the study, for example due to other health conditions. After agreeing to be assessed for participation, you will undergo a very detailed screening process at no cost to you, to determine your eligibility to participate in a study. 

How are clinical trials regulated?

All clinical trials are governed by rules set up by the federal government. Specialized training is required for all the investigators and coordinators who help conduct a clinical trial. The investigators who lead the clinical trials at CRND have been working on clinical trials for as long as 10 years.