Introduction
Another resource offered by the U.S. National Library of Medicine is the database of interventional and observational studies that can be found at http://www.clinicaltrials.gov. Interventional studies are also commonly called clinical trials, while observational studies do not involve any sort of intervention or treatment. Studies in all stages are listed on the website for anyone to view. If you are interested in finding out if a particular type of trial might be available in your area, there is an advanced search option under “Find a Study” where you can get as specific as you want with your parameters. Even if you don’t find an exact match, you can tweak your search terms and probably change the outcome of the search. Along the way you will be offered many links to studies with titles that give you a glimpse into the world of science and proof that “someone” is most definitely “doing something”!
Trials occur in phases, or stages, based on study objectives, participants, and other factors (observational studies do not operate in phases):
- Early Phase 1: exploratory studies conducted before the traditional trials begin, involve very small doses of the drug and make no claims of therapy or diagnosis of any disease
- Phase 1: studies focused on the safety of a drug, usually conducted with healthy volunteers, main goal is to study adverse events, their frequency, and how the body breaks down and gets rid of the drug
- Phase 2: studies focused on gathering preliminary data in patients with the disease, frequently involve some patients receiving a placebo, measuring safety and adverse events still a priority
- Phase 3: studies that receive the benefit of data from the first few phases of trials and involve more participants across specific populations at different dosages, measuring safety and efficacy of the drug still a priority
- Phase 4: trials that occur after FDA approval of the drug that continue to gather safety and effectiveness data to ensure optimal use of the drug
Summer, 2018
Pediatric Clinical Study; LINC5
University of California, San Francisco (UCSF) Updated September 2024
Do you have a child aged 6-17 years old with Cushing’s disease?
If so, your child may be able to participate in a research study to evaluate the effects and tolerability of osilodrostat (Isturisa) in children and adolescent patients with Cushing’s disease. Isturisa is currently FDA-approved for adults, and works by blocking an enzyme that helps make cortisol leading to less cortisol in the body.
Families who participate in clinical trials and other research studies contribute to science and learn more about their child’s condition under top doctors who run those trials, and findings from the research help future patients as well. Most trials for medications, including this one, also offer extension periods after the initial trial if the patient does well on the medication being studied and wishes to stay on it. Trial sponsors do everything they can to lessen the burden of participation including covering expenses like travel, meals, and lodging, and they will sometimes offer financial compensation for participation. This trial offers all of the above!
To learn more about this study being conducted at the University of California – San Francisco (UCSF), please contact Luis Gay, MPH, UCSF Pediatric Endocrinology, at 415-990-7296 or [email protected].
Click for more information: UCSF pediatric clinical trial flyer