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Phase 2 N=47 Randomized Quadruple-blind Treatment

N-Acetylcysteine for Neuroprotection in Parkinson's Disease

Parkinson Disease

Enrolled (actual)
47
Serious AEs
0.0%
Results posted
Apr 2018
Primary outcome: Primary: Change of Cerebral Glutathione Levels as Measured by Proton Magnetic Resonance Spectroscopy — 3.422; 3.334; 3.745; 4.282 Ratio

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
N-acetylcysteine (Drug); Placebo (Drug)
Age
Adult, Older Adult · 50+ yrs
Sex
All
Sponsor
Weill Medical College of Cornell University
Primary completion
Aug 2016

Outcome Measures

OutcomeResultp-value
PRIMARY
Change of Cerebral Glutathione Levels as Measured by Proton Magnetic Resonance Spectroscopy
3.422; 3.334; 3.745; 4.282; 4.415; 3.901
SECONDARY
Unified Parkinson's Disease Rating Scale (UPDRS) Parts I-V (Total Score Reported)
35.286; 31.500; 16.714; 1.000; 4.500; 3.143
SECONDARY
Mini Mental State Examination (MMSE)
29.571; 29.857; 29.571; 29.778; 29.625; 30.000
SECONDARY
Hamilton Depression Rating Scale (HAM-D)
3.714; 3.571; 1.857; 2.222; 1.875; 1.889
SECONDARY
9-Hole Peg Board Test (9-HPT)
37.026; 27.795; 28.694; 25.088; 24.593; 25.834
SECONDARY
10-Meter Walk Test
7.816; 7.289; 6.896; 7.253; 10.403; 7.141
SECONDARY
Beck Anxiety Inventory
7.000; 9.000; 6.333; 3.778; 3.750; 2.625
SECONDARY
Parkinson's Disease Quality of Life Questionnaire (PDQLQ)
134.333; 142.285; 159.833; 175.667; 171.625; 175.250

Summary

The overall objective of this developmental/exploratory study is to use noninvasive proton magnetic resonance spectroscopy (1H MRS) to assess (a) whether brain levels of the antioxidant glutathione (GSH) are decreased in vivo, as has been found in postmortem brain, in 30 patients with Parkinson's disease (PD) compared to matched controls; (b) whether GSH levels in PD brain increase significantly following 30 days of daily supplementation with 1800mg or 3600mg of N-acetylcysteine (NAC) compared to placebo and to baseline, and (c) whether any such increases in brain GSH would be dose-dependent and be associated with a change in the participants' oxidative stress profiles. In addition, a clinical assessment battery, including quantitative tests of motor function, will be performed to investigate potential associations between the NAC intervention, brain GSH levels, oxidative stress markers, and clinical presentation. If successful, this study will represent the first objective documentation of whether there is a GSH deficit in living PD brain that dietary NAC supplementation can mitigate, thereby providing a compelling justification for investigating such neuroprotective strategies in larger controlled clinical trials.

Eligibility Criteria

Inclusion Criteria

  • Diagnosis of idiopathic PD according to the United Kingdom Parkinson's Disease Society Brain Bank criteria (UKPDSBB) criteria (only for PD group
  • Age 50 to 75 years
  • Able to give informed consent for study participation
  • Not on any medication for PD (anticholinergic agents allowed)

Exclusion Criteria

  • Unable to give informed consent
  • Unable to undergo a brain MRI
  • PD duration ≥15 years
  • Receiving dopamine receptor blocking agents, including typical neuroleptics, prochlorperazine, and metoclopramide
  • Diagnosis of major depression or other axis I psychopathology
  • Modified Mini-Mental Status Exam (MMSE) ≤ 24/30
  • Diagnosis of chronic or persistent illnesses that could affect oxidative stress status, such as diabetes or congestive heart failure
  • Significant concomitant medical disease limiting life expectancy to less than 12 months from study inclusion
  • Diagnosis of primary mitochondrial disorder, epilepsy, stroke, multiple sclerosis or other neurodegenerative diseases such as Alzheimer's disease or ALS
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01470027). 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.

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