Does N-Acetylcysteine Decrease Spontaneous Oxidation of Central Neural Dopamine in Parkinson's Disease?

NCT ID: NCT03104725

Last Updated: 2021-11-04

Study Results

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-09-25

Study Completion Date

2020-02-27

Brief Summary

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Background:

Parkinsons disease (PD) causes slow movement, stiffness, and poor balance. Many symptoms are due to the loss of brain cells that make the brain chemical dopamine. The cells may be damaged by the breakdown of dopamine by a process called oxidation. The drug N-acetylcysteine (NAC) can act as an antioxidant. Researchers want to test if NAC can decrease the oxidation of brain dopamine in people with PD.

Objective:

To look at the effect of NAC on brain chemistry in people with PD.

Eligibility:

People ages 18 and older with PD that were diagnosed within the past 5 years. They must be taking a monoamine oxidase inhibitor.

Healthy volunteer participants ages 18 and older.

Design:

Participants will be screened with:

Medical history

Physical exam

Blood and urine tests

Participants will be hospitalized for 4 to 8 days.

On day 1, participants will have blood and urine tests. For several hours, they cannot eat or drink anything but water and their medications. Late in the morning they will have a meal.

About 2 hours later they will have a spinal tap (lumbar puncture). For this, a numbing medicine is injected into the back. A needle is inserted between the bones in the back to remove a small amount of fluid. The spinal tap may use x-rays to see inside the body.

After the spinal tap, they will start taking NAC by mouth.

They will take NAC twice a day for 2 more days.

On the next day, they will not eat until a meal in the late morning. They will take a final NAC dose.

About 2 hours later they will have a second spinal tap.

Healthy Volunteer (HV) participants will receive a spinal tap on day one, followed by a second spinal tap 48 hours after the first spinal tap. HV participants will not receive NAC.

Detailed Description

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Objective:

This study is to test whether N-acetylcysteine (NAC) inhibits the spontaneous oxidation of central neural dopamine as indicated by the cerebrospinal fluid (CSF) concentration of 5-S-cysteinyl-dopamine (Cys-DA) in patients with Parkinsons disease (PD).

Study population:

The study population comprises up to 35 participants with early (less than or equal to 5 years from diagnosis), mild, levodopa-untreated PD and up to 6 healthy volunteer participants. The PD participants will be on an inhibitor of monoamine oxidase (MAO) that is prescribed for their disease.

Design:

The study has a two groups employing a pretest-posttest design. Each participant undergoes a lumbar puncture (LP) as an inpatient at the NIH Clinical Center to obtain cerebrospinal fluid (CSF) for assays of Cys-DA, 3,4-dihydroxyphenylacetic acid (DOPAC), and related biochemicals. For PD participants, the second LP is done after the participant has taken at least 5 doses of NAC (2 grams orally twice per day). The LP takes place about 2 hours after the last NAC dose. For HV participants the second LP takes place approximately 48 hours after the first LP.

Outcome measures:

The main outcome measure is the CSF concentration of Cys-DA. Other outcome measures are levels of other catecholamine-related neurochemicals or of indices of oxidative stress. Depending on the results, an exploratory study may be done involving NAC at 1 gram orally twice per day.

Conditions

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Parkinson Disease Cerebrospinal Fluid

Keywords

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5-S-Cysteinyldopamine Cerebrospinal Fluid PARKINSONS DISEASE MONOAMINE OXIDASE INHIBITOR

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

BASIC_SCIENCE

Blinding Strategy

NONE

Study Groups

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Healthy Volunteers

HVs who undergo 2 LPs as inpatients, with 48 hours between LPs and no NAC treatment

Group Type EXPERIMENTAL

Lumbar Puncture

Intervention Type PROCEDURE

Each participant undergoes a baseline lumbar puncture (LP 1) as an inpatient at the NIH Clinical Center to obtain cerebrospinal fluid (CSF) for assays of Cys-DA, 3,4-dihydroxyphenylacetic acid (DOPAC), and related biochemicals. The second LP (LP 2) is completed after the PD participant has taken at least 5 doses of NAC (2 grams orally twice per day) and approximately 2 hours after the last NAC dose. For the HV participant, LP 2 occurs approximately 48 hours after LP 1 (no NAC administered).

Fluoroscopy

Intervention Type RADIATION

If needed for lumbar puncture

PD Patients

Patient undergoes a lumbar puncture (LP) as an inpatient at the NIH Clinical Center to obtain cerebrospinal fluid (CSF) for assays of Cys-DA, 3,4- dihydroxyphenylacetic acid (DOPAC), and related biochemicals. The second LP is done after the patient has taken at least 5 doses of NAC (2 grams orally twice per day).

Group Type EXPERIMENTAL

N-Acetylcysteine

Intervention Type DRUG

Oral

Lumbar Puncture

Intervention Type PROCEDURE

Each participant undergoes a baseline lumbar puncture (LP 1) as an inpatient at the NIH Clinical Center to obtain cerebrospinal fluid (CSF) for assays of Cys-DA, 3,4-dihydroxyphenylacetic acid (DOPAC), and related biochemicals. The second LP (LP 2) is completed after the PD participant has taken at least 5 doses of NAC (2 grams orally twice per day) and approximately 2 hours after the last NAC dose. For the HV participant, LP 2 occurs approximately 48 hours after LP 1 (no NAC administered).

Fluoroscopy

Intervention Type RADIATION

If needed for lumbar puncture

Interventions

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N-Acetylcysteine

Oral

Intervention Type DRUG

Lumbar Puncture

Each participant undergoes a baseline lumbar puncture (LP 1) as an inpatient at the NIH Clinical Center to obtain cerebrospinal fluid (CSF) for assays of Cys-DA, 3,4-dihydroxyphenylacetic acid (DOPAC), and related biochemicals. The second LP (LP 2) is completed after the PD participant has taken at least 5 doses of NAC (2 grams orally twice per day) and approximately 2 hours after the last NAC dose. For the HV participant, LP 2 occurs approximately 48 hours after LP 1 (no NAC administered).

Intervention Type PROCEDURE

Fluoroscopy

If needed for lumbar puncture

Intervention Type RADIATION

Eligibility Criteria

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Inclusion Criteria

* PD diagnosed within the past 5 years
* Taking a monoamine oxidase (MAO) inhibitor
* Able to provide consent
* At least18 years old

Exclusion Criteria

* Taking levodopa in any form
* Known allergy to NAC
* Already taking an anti-oxidant dietary supplement (e.g., Olive Leaf Extract, MitoQ)
* A condition that would increase risk from a lumbar puncture (e.g., symptomatic spinal stenosis or myoclonus)
* History of a post-spinal headache that required treatment with a blood patch
* On a prescribed anti-coagulant (e.g., Coumadin, Plavix)
* Pregnant or breast-feeding
* History of alcohol or drug abuse
* Any medical condition thatcould put subjects at increased risk. Potential participants are excluded who have evidence of bone marrow, liver, or kidney failure based on abnormal screening lab results.
* On a medication that could interfere with the scientific results. An example of an exclusionary drug is the catechol-O-methyltransferase inhibitor entacapone. Tricyclic anti-depressants are another type of exclusionary drug
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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David S Goldstein, M.D.

Role: PRINCIPAL_INVESTIGATOR

National Institute of Neurological Disorders and Stroke (NINDS)

Locations

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National Institutes of Health Clinical Center

Bethesda, Maryland, United States

Site Status

Countries

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United States

References

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Goldstein DS, Holmes C, Sullivan P, Jinsmaa Y, Kopin IJ, Sharabi Y. Elevated cerebrospinal fluid ratios of cysteinyl-dopamine/3,4-dihydroxyphenylacetic acid in parkinsonian synucleinopathies. Parkinsonism Relat Disord. 2016 Oct;31:79-86. doi: 10.1016/j.parkreldis.2016.07.009. Epub 2016 Jul 20.

Reference Type BACKGROUND
PMID: 27474472 (View on PubMed)

Goldstein DS, Kopin IJ, Sharabi Y. Catecholamine autotoxicity. Implications for pharmacology and therapeutics of Parkinson disease and related disorders. Pharmacol Ther. 2014 Dec;144(3):268-82. doi: 10.1016/j.pharmthera.2014.06.006. Epub 2014 Jun 16.

Reference Type BACKGROUND
PMID: 24945828 (View on PubMed)

Goldstein DS, Jinsmaa Y, Sullivan P, Holmes C, Kopin IJ, Sharabi Y. Comparison of Monoamine Oxidase Inhibitors in Decreasing Production of the Autotoxic Dopamine Metabolite 3,4-Dihydroxyphenylacetaldehyde in PC12 Cells. J Pharmacol Exp Ther. 2016 Feb;356(2):483-92. doi: 10.1124/jpet.115.230201. Epub 2015 Nov 16.

Reference Type BACKGROUND
PMID: 26574516 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

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Other Identifiers

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17-N-0076

Identifier Type: -

Identifier Source: secondary_id

170076

Identifier Type: -

Identifier Source: org_study_id