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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02046434
Recruitment Status : Active, not recruiting
First Posted : January 27, 2014
Last Update Posted : May 27, 2021
Sponsor:
Information provided by (Responsible Party):
University of Colorado, Denver

Tracking Information
First Submitted Date  ICMJE January 23, 2014
First Posted Date  ICMJE January 27, 2014
Last Update Posted Date May 27, 2021
Actual Study Start Date  ICMJE January 2014
Actual Primary Completion Date February 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 23, 2014)
Levels of alpha-synuclein in blood plasma [ Time Frame: 1 month ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phenylbutyrate Response as a Biomarker for Alpha-synuclein Clearance From the Brain
Official Title  ICMJE Phenylbutyrate Response As a Biomarker for Alpha-Synuclein Clearance From Brain
Brief 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.
Detailed Description This is a Phase I clinical trial of phenylbutyrate in 20 Parkinson patients and 20 age- and sex-matched normal control subjects to see if phenylbutyrate can increase the removal of alpha-synuclein from the brain into the bloodstream. All subjects will receive 20 grams/day of phenylbutyrate in the liquid form phenylbutyrate-triglyceride taken as one teaspoonful three times per day with meals. Blood will be drawn on two days prior to starting phenylbutyrate to measure alpha-synuclein concentrations. Phenylbutyrate-triglyceride will then be started and the change in plasma alpha-synuclein will be measured on day 1, 7, 14, and 21 days while taking phenylbutyrate. After 21 days, the drug will be stopped and a final blood sample will be measured at 28 days to see if plasma alpha-synuclein has fallen to its pre-phenylbutyrate level. No effects on Parkinson symptoms are expected during this short trial. Please note that although taking any type of Parkinson's drugs for symptomatic treatment disqualifies you, if you and your neurologist are willing and able to have you off Parkinson medication for six weeks before and during the trial, you may be eligible to participate.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Parkinson's Disease
Intervention  ICMJE Drug: Glycerol Phenylbutyrate
Other Name: Ravicti
Study Arms  ICMJE
  • Experimental: Parkinson's Diesase
    Participant will take Glycerol Phenylbutyrate, participant has Parkinson's Disease
    Intervention: Drug: Glycerol Phenylbutyrate
  • Experimental: Control
    Participant does not have Parkinson's Disease, Participant will be taking glycerol phenylbutyrate
    Intervention: Drug: Glycerol Phenylbutyrate
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: January 23, 2014)
40
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 2021
Actual Primary Completion Date February 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 21 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02046434
Other Study ID Numbers  ICMJE 13-2808
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party University of Colorado, Denver
Study Sponsor  ICMJE University of Colorado, Denver
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Curt R Freed, MD University of Colorado, Denver
PRS Account University of Colorado, Denver
Verification Date May 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP