Parkinson's Clinical Trial Companion Navigating Clinical Trials - Page 49

Intervention A potential therapy or treatment that is tested in clinical trials. These may include drugs, medical devices or procedures, and they may be investigational products or therapies that are already available. (See: repurposing.) Interventions also can also include non- invasive approaches, such as exercise or physical therapy. Interventional trial A type of trial in which participants receive an intervention (e.g., drug or surgical procedure) so that researchers can evaluate the effects of the intervention on certain symptoms or other features associated with a disease. See also: intervention Longitudinal study A study that follows participants over an extended period of time, often years or decades, and is generally observational in nature. This type of study is particularly useful for evaluating risk factors or progression of a disease. See also: observational study LRRK2 The LRRK2 gene directs the production of the LRRK2 protein kinase, an enzyme that modifies the function of other proteins. The LRRK2 gene is implicated in one to two percent of all Parkinson’s disease cases. Lumbar puncture (LP) A lumbar puncture (LP), or spinal tap, is a procedure where a small needle is inserted below the spinal cord between the bones of the lower back to obtain a small amount of spinal fluid for analysis. Magnetic Resonance Imaging (MRI) scan A Magnetic Resonance Imaging (MRI) scan uses magnetic waves to create detailed pictures of areas inside the body. MRIs are especially useful for imaging the brain, and give clues about structure but not function. Some forms of MRI are being looked at as possible biomarkers. See also: biomarker Multicenter trial Observational studies A clinical study in which participants’ health and other data is measured, but volunteers do not receive an intervention or drug. See also: intervention Open label Clinical trials in which both investigators and participants know which participants have been assigned the intervention or placebo. A clinical trial performed at more than one medical or research institution. See also: blinding Neuroprotective treatment A test or examination used to measure the effects of an intervention on certain symptoms or other features associated with a disease. Investigators decide on the measures that they are interested in evaluating before the trial or study begins. Every interventional study has a primary outcome measure, which is most important for evaluating the effect of the intervention. Studies also may include secondary outcome measures, which are not as important but are still of interest in evaluating the effect. A therapy that guards against death or damage of the dopamine cells in the brain that are at risk of being lost in Parkinson’s disease (PD). There is currently no approved neuroprotective therapy for Parkinson’s, but research in this area is ongoing. These types of therapies could theoretically be used in people with early signs of PD or even those who are at risk. Neuroregenerative treatment A therapy that stimulates regrowth of dopamine-producing cells in the brain. There is currently no approved neurorege nerative therapy for Parkinson’s, but research in this area is ongoing. New Drug Application (NDA) A new drug application (NDA) is a formal request from a drug sponsor to the U.S. FDA to ask for approval of a new drug. Data from pre-clinical research and all phases of human clinical trials are submitted as part of the NDA. See also: U.S. Food and Drug Administration Outcome measure Patient-reported outcomes (PROs) Data that is provided directly by participants. Patient-reported outcomes (PROs) complement traditional measures used during in-person clinical trial and study visits to give researchers a more complete picture of disease. Placebo A substance or device that does not contain active ingredients but is made to look, feel and taste just like the actual drug or therapy being studied so that all participants have a similar research experience. See also: placebo-controlled Glossary 47