What is a clinical trial?
A clinical trial is a research study that looks into the safety and usefulness of a drug in improving symptoms or slowing, stopping, or reversing the progression of a disease. Most drugs tested in clinical trials are not yet available to the public (Phase I, II, III studies) while some studies involve medications that are already available (Phase IV studies).
What happens during the study if I decide to participate?
Each clinical trial requires specific criteria for enrollment. You may express your desire to inquire about a particular study to any of our clinical staff. Your medical history assists us to determine your eligibility. Then the consent form, which outlines your rights and responsibilities as a research subject and the potential benefits and risks in study participation, will be carefully explained to you. Once you decide to participate, the study coordinator will contact you and give you the schedule and the details you will need to know for each visit. You have the right to quit at anytime without prejudice to you or the care you receive.
Do I get compensated for participating in a clinical trial?
Some studies that require prolonged visits or an increased number visits, may be compensated for the gasoline, meals, lodging and parking expenses. However, in general, participating in a research study is a selfless voluntary action.
Why should I participate in a clinical trial?
People participate for various reasons. Most do because they want to contribute to the therapeutic advancement of their illness. Some participate because the medication being tested is not yet available in drug stores and it may potentially improve their symptoms or slow their disease. Some patients participate for the free comprehensive physical and neurological evaluations and screening laboratory tests that are required in the study. Patients often enjoy being in a clinical trial because it may serve as a way of learning about their illness. Even though we treat all our patients with the best available care, whether or not they choose to join a clinical trial, participants get extra benefits from enrolling. These benefits include closer monitoring of their symptoms, more one-on-one interaction with their doctor and nurse, and increased learning about their disease.
Gene Therapy FAQ
The National Organization for Rare Disorders (NORD) launched Gene Therapy: Your Questions Answered, a new video from its RareEDU educational program aimed at addressing some of the questions, hopes and concerns that patients and caregivers, across many different diseases, have about gene therapy. Emma Crowley, our patient advocate, and Dr. Cristina Liberati from the Powell Gene Therapy Center at the University of Florida collaborated with NORD to craft accurate answers to some of the most frequently asked questions about gene therapy. The video was made possible by a donation from Spark Therapeutics and NORD is solely responsible for the content. It is available for viewing by clicking the button below.
Questions covered in Gene Therapy: Your Questions Answered include:
- Is gene therapy a cure?
- Why is gene therapy getting so much attention?
- Why does it cost so much?
- Will insurance pay for it?
- What is the difference between gene therapy and gene editing?
Interested in our pre-clinical research?
The Powell Gene Therapy Center (PGTC) at the University of Florida
The primary mission of the Gene Therapy Center at the University of Florida is to merge molecular genetics research and health care delivery by developing new therapeutic strategies for the treatment of human diseases that involve gene transfer. The idea of gene therapy is a logical and natural progression of the last 20 years of research in medical genetics and molecular biology. PGTC has been instrumental in the development of newer, safer agents for the delivery of therapeutic genes to patients with genetic diseases, such as cystic fibrosis (CF) and alpha 1-antitrypsin deficiency (AAT-D).