Parkinson's Clinical Trial Companion Navigating Clinical Trials - Page 48

Glossary European Medicines Agency ( EMA) Gene Idiopathic Parkinson’s disease A decentralized agency of the European Union (EU) responsible for the scientific evaluation, supervision, safety and monitoring of medicines in the EU. The material of heredity, passed down through the generations from parents to children. These inherited bits of DNA determine many of the body’s traits — visible features such as eye color, as well as ones that can’t be seen, including an individual’s risk of a particular disease. The most common form of Parkinson’s, accounting for upwards of 90 percent of cases. Idiopathic, or sporadic, Parkinson’s typically does not run in families and is likely caused by a combination of genetic and environmental factors. Genetic counselor Inclusion criteria Exclusion criteria Factors that prevent a person from participating in a specific clinical trial or study. Familial Parkinson’s disease A type of Parkinson’s that runs in families and is thought to have a primarily genetic cause. Familial Parkinson’s disease accounts for less than 10 percent of PD cases worldwide. See also: idiopathic Parkinson’s disease Gaucher disease A rare condition that causes fatty substances to build up and organs to swell. This disease develops in people who carry two copies of the mutated GBA gene. People with this mutation do not produce enough glucocerebrosidase, an enzyme that breaks down a fatty chemical called glucocerebroside. A health professional with expertise in medical genetics and counseling who provides education and emotional support to people considering or undergoing genetic testing. Genetic mutation A permanent change in the sequence of a gene that can affect health or risk of disease. Genetic testing A type of medical test that identifies changes in genetic material. Genetic tests can evaluate a suspected genetic condition to help determine a person’s chance of developing or passing on a genetic disorder. See also: GBA Hoehn and Yahr ( H&Y) s cale GBA The Hoehn and Yahr (H&Y) scale divides Parkinson’s into stages based on the severity of motor symptoms. Clinical trials often include H&Y stages as part of their eligibility criteria so that they can ensure that the intervention evaluated will include people with the right symptoms. The GBA gene directs the production of the glucocerebrosidase protein, which breaks down substances called glycolipids. Mutations in the GBA gene are the most common genetic risk factor associated with Parkinson’s, and GBA mutations may lead to build-up of alpha- synuclein protein clumps. See also: alpha-synuclein 46 Navigating Clinical Trials: A Guide for Parkinson’s Patients and Families See also: familial Parkinson’s disease Factors that need to be met to qualify a person to participate in a clinical trial or study. Informed consent A process used to educate potential participants about the possible benefits and risks of a specific clinical trial or study. Prior to enrolling, all study participants must sign an informed consent document that explains the details of the trial or study and the rights and responsibilities of the participant. Institutional Review Board ( IRB) An independent committee of scientists, doctors and others (usually at least one “non-scientific” person who represents the patient voice) that evaluates and approves each study’s protocol and informed consent document, and monitors ongoing study activities. The Institutional Review Board (IRB) is in place to protect the rights and welfare of people participating in a study.