A clinical trial (also known as medical research, clinical study or drug trial) is a study designed to answer questions about the safety and effectiveness of new medical interventions including ways to screen, diagnose, treat, or prevent disease. Carefully conducted clinical trials are considered to be the fastest and safest way to find treatments that work in people.
Every clinical trial follows a pre-defined protocol that includes clear criteria for who is eligible to participate, detailed information about the purpose of the study, who is conducting it and the exact procedures, risks and potential benefits. Researchers will present essential information about the study to patients considering participation and should provide time to answer any questions. This information will also be presented in writing for participants to review and sign as part of the informed consent process for joining the study.
Clinical trials of new drugs take place in four phases which typically extend over many years:
Phase I studies are the first phase of testing a new drug or treatment in human subjects. These studies are conducted in a very small group of (usually) healthy volunteers. The goal of Phase I is to assess the safety and dosing of the drug by observing how it is metabolised in the human body.
Phase II studies test the drug in a larger group of both healthy volunteers and patients. The goal is to determine the drug’s effectiveness and to identify any common side effects or risks.
Phase III studies are usually conducted in a much larger group of people across multiple centers. During Phase III patients may be randomly assigned to two groups: one group (the test group) receives the new drug and the other group (the control group) receives a “placebo,” or sugar pill that looks exactly like the drug but has no effect. In double-blind studies neither the patient nor the physician knows which pill is which. This eliminates the risk of unintentional bias on the part of the physician who will be observing and recording any change in the patient’s condition throughout the study. Phase III trials are considered the definitive test to determine the drug’s effectiveness, side effects and proper dosing.
Phase IV studies takes place after the drug has been approved for use. They continue to monitor the safety and effects of the drug long-term.
What to expect
Not everyone who is interested in participating in a specific clinical trial will be eligible. Each study protocol has strict inclusion and exclusion criteria to ensure that the results can be interpreted clearly. If a person is eligible, the research team will explain the study in detail including potential risks and benefits and what the volunteer must do if he or she becomes involved. After careful consideration, the interested volunteer will sign the informed consent and be enrolled.
Members of the research team will evaluate the participant’s health at the start of the trial and give the person specific instructions about participating. The participant will be monitored carefully during the study according to the protocol, and typically will have some continued contact with the research team after the trial is finished. Usually, there is no cost to the volunteer for study-related services; travel expenses may be covered as well.
How to participate
People interested in participating in clinical trials should make sure they understand what is involved in volunteering for clinical research as well as the details of the specific study for which they are eligible.
There are various ways to learn about what clinical research is being conducted in frontotemporal degeneration. The most comprehensive is ClinicalTrials.gov, a registry maintained by the U.S. National Institutes of Health (NIH) of federally and privately funded clinical research. A search feature lets you look specifically for studies in FTD
ClinicalTrials.gov – provides patients, family members, and members of the public easy and free access to information on clinical studies for a wide range of diseases and conditions. Enter “frontotemporal degeneration” and any other specific search criteria to view a full list of all registered trials, eligibility criteria, participating medical centers and contact information for each.
NIH Clinical Center – conducts research in a wide range of diseases, including frontotemporal dementia. The Clinical Center’s website has created this new website to help people learn more about clinical trials, why they matter and how to participate.
Research has shown that some of the greatest challenges to recruitment of volunteers are the lack of general knowledge about what trials involve, where they are carried out, and who may participate. The new, centralized resource will make it easier for the public and health professionals to learn about clinical trials and how people can participate.
Visitors to the site will find information about the basics of clinical trial participation . experiences from clinical trial volunteers and explanations from researchers . links on how to search for a trial or enroll in a research matching program In addition, health care professionals can read about evidence-based strategies for talking with patients about trials, and find audience-tested posters to help promote trials in clinics and offices.
Webinar Training on FTD Clinical Trials
Recorded: May 13, 2013
Susan Dickinson, CGC, MS
Jill Shapira, PhD, RN
Training was provided to FTD support group facilitators for continuing education. The goal of this session is to educate and empower people with FTD and their families as they face increasing opportunities to participate in research that will lead to the first approved drugs for FTD. Click to access the archived webinar, the handout and slides from the presentation.