Phase 1
N=9
Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease
Parkinson's Disease · Gaucher's Disease
Bottom Line
View on ClinicalTrials.gov: NCT01427517 ↗Enrolled (actual)
9
Serious AEs
0.0%
Results posted
Aug 2013
Primary outcome: Primary: Brain GSH — 55; 41; 34 percent increase from baseline
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- N-acetylcysteine (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- University of Minnesota
- Primary completion
- Dec 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Brain GSH |
55; 41; 34 | — |
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.
Eligibility Criteria
Inclusion Criteria
- All participants must be 18 years or older.
- 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.
- Individuals with medically stable Parkinson's disease (in the opinion of the investigator).
- All participants must not have taken antioxidants coenzyme Q-10, vitamin C, or vitamin E for 3 weeks prior to the study.
- Absence of dementia in all subjects, as determined by pre-scanning cognitive assessment.
- Control subjects who are able to undergo MRS
Exclusion Criteria
- Inability to undergo MRI scanning without sedation
- Medically unstable conditions in any group as determined by the investigators
- Pregnant or lactating or those women of child-bearing age that are not using acceptable forms of contraception
- 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
- Unable to adhere to study protocol for whatever reason
Data sourced from ClinicalTrials.gov (NCT01427517). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.