What is a clinical trial?

Clinical trials are research studies that explore whether a medical strategy, treatment, or device is safe and effective for humans. These studies also may show which medical approaches work best for certain illnesses or groups of people. Clinical trials produce the best data available for health care decision making.

The purpose of clinical trials is research, so the studies follow strict scientific standards. These standards protect patients and help produce reliable study results.

Clinical trials are a key research tool for advancing medical knowledge and patient care. Clinical research is done only if doctors don’t know:

  • whether a new approach works well in people and is safe and
  • which treatments or strategies work best for certain illnesses or groups of people.

Some clinical trials show a positive result. The results from other clinical trials show what doesn’t work or may cause harm.

Each clinical trial has a master plan called a protocol. This plan explains how the trial will work. The trial is led by a principal investigator (PI), who often is a doctor. The PI prepares the protocol for the clinical trial.

The protocol outlines what will be done during the clinical trial and why. Each medical center that does the study uses the same protocol. (Source: National Institute of Health)

Is There a Clinical Trial for My Gene?

ClinicalTrials.gov is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world.

The website is supported by the United States National Institute of Health (NIH). It lists more than 200,000 studies in all 50 U.S. states and in 190 countries worldwide.

It is very important that before participating in any trial, you confirm it is an approved trial by searching for it on clinicaltrials.gov and discussing the trial with your physician or specialist. You should never participate in any trial that is not listed on the government website and your doctor has not reviewed and discussed with you.

My Retina Tracker will provide you with the most up-to-date information about research that may be of specific interest to you, and other people with inherited retinal degenerative diseases, including clinical trials to evaluate possible new treatments.

Scientists studying inherited retinal degenerative diseases need accurate, firsthand information from many people to understand how each inherited retinal degenerative disease affects people, information only you and your doctor can provide. When scientists are ready to start research studies or clinical trials, they can use information in the Registry to help identify people that may be eligible to participate in studies. If an affected individual looks like a good match for a scientist’s research or a clinical trial, the scientist or clinical trial coordinator will contact the FFB Registry. Then a member of the FFB Registry staff, or someone working with the Registry such as genetic counselor, will contact you, and other individuals identified by the investigator. Scientists will not contact you directly unless you give the FFB permission for them to do so.
(Source: My Retina Tracker)