Clinical Trials
What is a Clinical Trial?
Clinical trials are rigorous research studies in humans that determine whether a treatment or device is safe and effective. Clinical trials follow strict scientific standards. These standards protect patients and help produce reliable study results.
In the US, clinical trials are authorized and regulated by the US Food & Drug Administration (FDA). In Europe, clinical trial oversight is provided by the European Medicines Agency (EMA). While the FDA and EMA are the world’s most prominent and respected regulatory agencies, there are several other regulatory agencies in individual countries.
Clinical trials typically run for six to 10 years and cost, at a minimum, tens of millions of dollars.
Clinical trials for rare diseases like LCA typically enroll a few dozen patients. Clinical trials for more common conditions may enroll hundreds or thousands of patients.
When a company or research group decides to launch a clinical trial, they must file an Investigational New Drug (IND) application with regulators. The IND extensively documents the lab research conducted to show a treatment’s safety and efficacy in non-human studies. It also outlines the trial protocol and clinical manufacturing practices for the emerging therapy.
Clinical trials are conducted in three phases, though phases are often combined for gene and cell therapies for rare diseases. Safety monitoring is paramount and evaluated in all phases.
Phase 1 evaluates preliminary safety. Phase 2 explores different doses and preliminary efficacy. Phase 3, also known as pivotal or confirmatory, often takes place at multiple sites. If the Phase 3 safety and data are favorable, the therapy developer will likely seek marketing approval for the treatment and will either submit a New Drug Application (NDA) to the FDA for drugs or a Biologics License Application (BLA) for gene, protein, or cell therapies.
Clinical trials are an important research tool for advancing medical knowledge and patient care, even if a trial doesn’t lead to an approved therapy.
Is There a Clinical Trial for Me?
ClinicalTrials.gov is a searchable database of publicly and privately supported human studies in the US and many other countries. The website is supported by the United States National Institute of Health (NIH).
Most but not all clinical trials on clinicaltrials.gov are properly regulated. You should never participate in any trial that is not monitored by a regulatory agency (e.g., FDA or EMA). Do not participate in a clinical trial that charges a fee for the treatment. Most clinical trials will pay for travel and accommodations.
MyRetinaTracker.org is a free and secure patient registry hosted by the Foundation Fighting Blindness for people with inherited retinal diseases. Many (but not all) therapy developers use the registry to help identify people who may be eligible to participate in clinical studies. If an affected individual appears to be a good match for a therapy developer’s research or a clinical trial, a member of the Foundation’s Registry staff will contact the individual. Therapy developers do not have access to patients’ personal information and will not contact you directly unless you give the Foundation permission for them to do so.
(Source: My Retina Tracker)