Repeated-Dose Oral N-acetylcysteine for the Treatment of Parkinson's Disease

The investigators are interested in evaluating the use of oral N-acetylcysteine (NAC) as therapy for Parkinson's Disease (PD) and measuring changes in brain and blood chemistry.

Study Overview

• Status: Completed
• Conditions: Parkinson Disease
• Intervention / Treatment: Drug: N-acetylcysteine capsule

Detailed Description

The investigators are interested in evaluating the use of oral N-acetylcysteine (NAC) as therapy for Parkinson's Disease (PD) and measuring changes in brain and blood chemistry. The investigators' hypothesis is that repeated oral dosing of NAC will result in increased brain and red blood cell GSH concentrations in individuals with Parkinson's disease (PD) and healthy controls. The specific aims are to 1.) measure plasma and blood (red blood cell) glutathione (GSH) and ratio of reduced GSH to oxidized glutathione (GSSG) in individuals with PD and healthy controls at baseline and after four weeks of repeated high doses of oral NAC (6000 mg/day) and 2) measure cortical GSH levels (as ascertained through MRS) in those with PD and healthy controls at baseline and four weeks during repeated high doses of oral NAC (6000 mg/day) simultaneously with Aim 1. This study will combine information from a medical history, a physical examination, and disease rating scales with results obtained using Magnetic Resonance Spectroscopy (MRS) brain scans and pharmacokinetic studies from blood samples. This research will require 2 visits, one at baseline and one after approximately 28 days of therapy. Participants will provide their medical history during the first visit, and undergo a physical examination and rating scale each visit (duration: 1 hour). The first visit including the brain scans and blood collection will require approximately 3 hours of time. The brain scan and pharmacokinetic studies for the second visit will require approximately 8 hours of time. In total the second visit will take roughly 9 hours.

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 NAC, glutathione, coenzyme Q-10, vitamin C, or vitamin E for 3 weeks prior to the study
5. Absence of dementia in all subjects

Exclusion Criteria:

1. Inability to undergo MRI scanning without sedation and other MRI counterindications, such as metal in the body (see section 7.3)
2. Medically unstable conditions 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: N-acetylcysteine; Subjects will be provided 6000 mg/day of N-acetylcysteine (capsule) to be divided into 2 equal daily doses and taken orally for approximately 28 days	Drug: N-acetylcysteine capsule; N-acetylcysteine capsule; Other Names: NAC

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Glutathione (GSH) Brain Levels	GSH levels in brain of all subjects at baseline and post-NAC (n-acetylcysteine) dosing as measured by magnetic resonance spectroscopy (MRS)	pre-dose and after approximately 28 days of treatment

Collaborators and Investigators

• Sponsor: University of Minnesota
• Collaborators: Parkinson's Disease Society of the United Kingdom
• Investigators: Principal Investigator: Paul Tuite, MD, University of Minnesota; Principal Investigator: Lisa Coles, PhD, University of Minnesota

Study record dates

Study Major Dates

• Study Start: September 1, 2014
• Primary Completion (Actual): September 1, 2015
• Study Completion (Actual): September 1, 2015

Study Registration Dates

• First Submitted: August 4, 2014
• First Submitted That Met QC Criteria: August 7, 2014
• First Posted (Estimate): August 8, 2014

Study Record Updates

• Last Update Posted (Actual): May 16, 2019
• Last Update Submitted That Met QC Criteria: April 23, 2019
• Last Verified: April 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Parkinson Disease; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Antiviral Agents; Protective Agents; Respiratory System Agents; Antioxidants; Antidotes; Free Radical Scavengers; Expectorants; Acetylcysteine; N-monoacetylcystine
• Other Study ID Numbers: 1406M51207; 123269 (Other Identifier: FDA)