Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease

NCT01427517 | Completed Phase 1 Results DMC

• 2 other identifiers: FWA00000312-2U54NS065768
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Parkinson's Disease; Gaucher's Disease

Keywords

Parkinson's disease; Gaucher's disease; Control

Outcome Measures

Primary Outcomes (1)

• Brain GSH

  change in brain GSH levels from baseline to post-NAC administration (90 - 110 minutes) in all subjects

  Baseline and up to 110 minutes post-NAC administration

Study Arms (3)

NAC in PD

EXPERIMENTAL

single intravenous administration of N-acetylcysteine in PD patients

Drug: N-acetylcysteine

NAC in GD

EXPERIMENTAL

single intravenous administration of N-acetylcysteine in GD patients

Drug: N-acetylcysteine

NAC in controls

EXPERIMENTAL

single intravenous administration of N-acetylcysteine in control subjects

Drug: N-acetylcysteine

Interventions

N-acetylcysteine DRUG

Single, intravenous administration of N-acetylcysteine

Also known as: NAC

NAC in GD; NAC in PD; NAC in controls

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Minnesota: lead
• Rare Diseases Clinical Research Network: collaborator
• National Center for Advancing Translational Sciences (NCATS): collaborator
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator
• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): collaborator

Study Sites (1)

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Related Publications (1)

• Holmay MJ, Terpstra M, Coles LD, Mishra U, Ahlskog M, Oz G, Cloyd JC, Tuite PJ. N-Acetylcysteine boosts brain and blood glutathione in Gaucher and Parkinson diseases. Clin Neuropharmacol. 2013 Jul-Aug;36(4):103-6. doi: 10.1097/WNF.0b013e31829ae713.

  PMID: 23860343 DERIVED

MeSH Terms

Conditions

Parkinson Disease; Gaucher Disease

Interventions

Acetylcysteine

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Cysteine; Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Mary Holmay
• Organization: University of Minnesota

holma101@umn.edu

612-812-5854

Study Officials

• Paul Tuite, MD

  University of Minnesota

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 30, 2011
• First Posted: September 1, 2011
• Study Start: July 1, 2011
• Primary Completion: December 1, 2012
• Study Completion: December 1, 2012
• Last Updated: November 1, 2019
• Results First Posted: August 2, 2013

Record last verified: 2019-10

Locations

US (1)