Phenylbutyrate Response as a Biomarker for Alpha-synuclein Clearance From the Brain
Phenylbutyrate Response As a Biomarker for Alpha-Synuclein Clearance From Brain
1 other identifier
interventional
38
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Jan 2014
Longer than P75 for phase_1
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 1, 2014
CompletedFirst Submitted
Initial submission to the registry
January 23, 2014
CompletedFirst Posted
Study publicly available on registry
January 27, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
September 1, 2019
CompletedApril 25, 2022
April 1, 2022
5.7 years
January 23, 2014
April 15, 2022
Conditions
Outcome Measures
Primary Outcomes (1)
Levels of alpha-synuclein in blood plasma
1 month
Study Arms (2)
Parkinson's Diesase
EXPERIMENTALParticipant will take Glycerol Phenylbutyrate, participant has Parkinson's Disease
Control
EXPERIMENTALParticipant does not have Parkinson's Disease, Participant will be taking glycerol phenylbutyrate
Interventions
Eligibility Criteria
You may qualify if:
- Idiopathic Parkinson's disease with mild symptoms;
- May be on treatment with dopamine agonists provided that the treating neurologist agrees:
- that the drugs can be stopped for at least three weeks prior to participating in the phenylbutyrate study, and
- 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.
You may not qualify if:
- Pregnant women;
- Current treatment with:
- L-3,4-dihydroxyphenylalanine (L-DOPA);
- monoamine oxidase (MAO) inhibitors,
- catechol-O-methyl transferase (COMT) inhibitors;
- histone deacetylase (HDAC) inhibitors;
- prednisone or other corticosteroids, or
- probenecid.
- Severe cardiopulmonary disease such as:
- congestive heart failure, or
- emphysema requiring supplemental oxygen;
- Renal disease with serum creatinine greater than 2.5;
- History of:
- depression in the prior year;
- epilepsy;
- +4 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
University of Colorado Denver Anschutz Medical Center
Aurora, Colorado, 80045, United States
Related Publications (1)
Wen ZQ, Lin J, Xie WQ, Shan YH, Zhen GH, Li YS. Insights into the underlying pathogenesis and therapeutic potential of endoplasmic reticulum stress in degenerative musculoskeletal diseases. Mil Med Res. 2023 Nov 9;10(1):54. doi: 10.1186/s40779-023-00485-5.
PMID: 37941072DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Curt R Freed, MD
University of Colorado, Denver
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 23, 2014
First Posted
January 27, 2014
Study Start
January 1, 2014
Primary Completion
September 1, 2019
Study Completion
September 1, 2019
Last Updated
April 25, 2022
Record last verified: 2022-04