Overview
Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease
Status:
Completed
Completed
Trial end date:
2012-12-01
2012-12-01
Target enrollment:
0
0
Participant gender:
All
All
Summary
The investigators are interested in determining if the investigators are able to detect changes in brain chemistry using Magnetic Resonance Spectroscopy (MRS) in individuals with Parkinson's disease (PD), those with Gaucher's disease (GD), and those without neurological disorders (healthy controls) when they are given the antioxidant N-acetylcysteine (NAC). This study will combine information from a medical history, a physical examination and disease rating scales with results obtained using MRS brain scans and pharmacokinetic studies from blood samples. This research will require 1 visit that will require about 4 to 5 hours of time. During this study, participants will provide their medical history, be examined and undergo a rating scale for about one hour; the brain scan and pharmacokinetic studies will require 1.5-2 hours of time; in total the study will take about 4-5 hours.Phase:
Phase 1Accepts Healthy Volunteers?
Accepts Healthy VolunteersDetails
Lead Sponsor:
University of Minnesota
University of Minnesota - Clinical and Translational Science InstituteCollaborators:
National Center for Advancing Translational Science (NCATS)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
National Institute of Neurological Disorders and Stroke (NINDS)
Rare Diseases Clinical Research NetworkTreatments:
Acetylcysteine
N-monoacetylcystine
Criteria
Inclusion Criteria:1. All participants must be 18 years or older.
2. All enrollees must understand and cooperate with requirements of the study in the
opinion of the investigators and must be able to provide written informed consent.
3. Individuals with medically stable Parkinson's disease (in the opinion of the
investigator).
4. All participants must not have taken antioxidants coenzyme Q-10, vitamin C, or vitamin
E for 3 weeks prior to the study.
5. Absence of dementia in all subjects, as determined by pre-scanning cognitive
assessment.
6. Control subjects who are able to undergo MRS
Exclusion Criteria:
1. Inability to undergo MRI scanning without sedation
2. Medically unstable conditions in any group as determined by the investigators
3. Pregnant or lactating or those women of child-bearing age that are not using
acceptable forms of contraception
4. Diagnosis of asthma that is presently being treated with ANY medication, or past
history of asthma/bronchospasm resulting in an emergency room visit, hospitalization
or treatment
5. Unable to adhere to study protocol for whatever reason