Parkinson's DiseasePhase 1NCT02046434
Phenylbutyrate Response as a Biomarker for Alpha-synuclein Clearance From the Brain
A Phase 1 study testing Glycerol Phenylbutyrate in people with Parkinson's Disease, run by University of Colorado, Denver at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 21 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- Glycerol Phenylbutyrate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2014to Sep 2019
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 to 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
Show all 19Show fewer
- 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;
- stroke;
- prior brain surgery;
- dementia, or
- psychosis.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Colorado Denver Anschutz Medical Center
Aurora, Colorado, 80045
About this study
Official title: Phenylbutyrate Response As a Biomarker for Alpha-Synuclein Clearance From Brain
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.
Read the 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.
- Conditions
- Parkinson's Disease
- Being tested
- Glycerol Phenylbutyrate
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Glycerol PhenylbutyrateDrug
Also called Ravicti
| Study group | Role | What they receive |
|---|---|---|
| Parkinson's Diesase | Experimental | Participant will take Glycerol Phenylbutyrate, participant has Parkinson's Disease Receives: Drug: Glycerol Phenylbutyrate |
| Control | Experimental | Participant does not have Parkinson's Disease, Participant will be taking glycerol phenylbutyrate Receives: Drug: Glycerol Phenylbutyrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Levels of alpha-synuclein in blood plasma
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2014 (estimated)
- Study startJan 2014
- Primary completionSep 2019
- Study completionSep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- More trials run by University of Colorado, DenverThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Colorado, Denver
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Curt R Freed, MD, Principal investigator, University of Colorado, Denver
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02046434
- Sponsor's protocol ID
- 13-2808
- Status last verified
- Apr 2022
- First submitted
- 23 Jan 2014
- Last updated
- 25 Apr 2022
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 37941072
Study at a glance
From the registry record, last updated by the sponsor on 25 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phenylbutyrate Response As a Biomarker for Alpha-Synuclein Clearance From Brain
- ClinicalTrials.gov ID
- NCT02046434
- Acronym / sponsor ID
- 13-2808
- Conditions
- Parkinson's Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- University of Colorado, DenverUniversity, hospital or other
- Study start
- Jan 2014 (actual)
- Primary completion
- Sep 2019 (actual)
- Study completion
- Sep 2019 (actual)
- First posted
- 27 Jan 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Parkinson's Diesase | Experimental | Drug: Glycerol PhenylbutyrateParticipant will take Glycerol Phenylbutyrate, participant has Parkinson's Disease |
| Control | Experimental | Drug: Glycerol PhenylbutyrateParticipant does not have Parkinson's Disease, Participant will be taking glycerol phenylbutyrate |
Who may qualify
- Ages
- 21 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Idiopathic Parkinson's disease with mild symptoms;
- May be on treatment with dopamine agonists provided that the treating neurologist agrees:
- Age and sex matched normal control subjects from spouses and the general population;
- In good general health;
- Controlled hypertension, or
Key exclusion criteria (19 listed)
- Pregnant women;
- Current treatment with:
- Severe cardiopulmonary disease such as:
- Renal disease with serum creatinine greater than 2.5;
- History of:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Glycerol PhenylbutyrateAlso: Ravicti | Drug | Control, Parkinson's Diesase |
Primary outcome
Levels of alpha-synuclein in blood plasma
Time frame: 1 month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Apr 2022.
- Derived from this study: PubMed 37941072Wen 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Curt R Freed, MD principal investigator
University of Colorado, Denver
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Glycerol Phenylbutyrate in people with Parkinson's Disease, run by University of Colorado, Denver at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Levels of alpha-synuclein in blood plasma (time frame: 1 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02046434, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Apr 2022.