Phenylbutyrate Response as a Biomarker for Alpha-synuclein Clearance From the Brain

• Status: Active, not recruiting
• Trial end date: 2021-10-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase I clinical trial of the FDA approved drug Glycerol Phenylbutyrate to see if phenylbutyrate can increase the removal of alpha-synuclein from the brain into the bloodstream. Alpha-synuclein forms abnormal protein deposits in dopamine neurons and is believed to cause the death of brain cells, leading to Parkinson's Disease.

• Phase: Phase 1
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: University of Colorado, Denver
• Treatments: 4-phenylbutyric acid; Glycerol

Criteria

Inclusion Criteria:

• Idiopathic Parkinson's disease with mild symptoms;

• May be on treatment with dopamine agonists provided that the treating neurologist agrees:

1. that the drugs can be stopped for at least three weeks prior to participating in the phenylbutyrate study, and

2. for the 4-week duration of the study.

• Age and sex matched normal control subjects from spouses and the general population;

• In good general health;

• Controlled hypertension, or

• Controlled hypercholesterolemia with medication.

Exclusion Criteria:

• Pregnant women;

• Current treatment with:

1. L-3,4-dihydroxyphenylalanine (L-DOPA);

2. monoamine oxidase (MAO) inhibitors,

3. catechol-O-methyl transferase (COMT) inhibitors;

4. histone deacetylase (HDAC) inhibitors;

5. prednisone or other corticosteroids, or

6. probenecid.

• Severe cardiopulmonary disease such as:

1. congestive heart failure, or

2. emphysema requiring supplemental oxygen;

• Renal disease with serum creatinine greater than 2.5;

• History of:

1. depression in the prior year;

2. epilepsy;

3. stroke;

4. prior brain surgery;

5. dementia, or

6. psychosis.