Safety and Biomarker Study of PTC-589 in Participants With Parkinson's Disease

April 6, 2022 updated by: Edison Pharmaceuticals Inc

A Phase 2A Safety and Biomarker Study of EPI-589 in Mitochondrial Subtype and Idiopathic Parkinson's Disease Subjects

Open-label study with 30-day run-in phase and adaptive design component to include more participants if deemed appropriate by investigators.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This is a within-subject, controlled open-label study seeking to determine if PTC-589 can alter the biochemical signature of Parkinson's disease as assessed by peripheral blood biomarkers, central nervous system (CNS) biomarkers, and urine biomarker analysis. In addition, data on a number of disease-relevant clinical measures will be collected.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
44

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Tuebingen, Germany, 72074
        • DZNE Site
  • United Kingdom
      • London, United Kingdom, NW3 2PF
        • University College of London,Dept. of Clinical Neuroscience
  • United States
    • California
      • Los Angeles, California, United States, 90048
        • Cedar's Sinai
      • San Francisco, California, United States, 94115
        • University of California, San Francisco
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Beth Israel Deaconess Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

17 years to 71 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Hoehn and Yahr stage ≤3.0
  • Ambulatory with or without assistance
  • Sexually active fertile participants and their partners must agree to use medically accepted methods of contraception (such as, hormonal methods, including oral, subcutaneous, and intrauterine; barrier methods, including male condom, female condom, or diaphragm with spermicidal gel) during the course of the study and for 3 months after the last dose of study treatment.
  • Willingness and ability to comply with study procedures
  • If on medications for Parkinson's disease drugs, then medication regimen must be stable for 60 days prior to enrollment
  • Abstention from use of other investigative or non-approved drugs for the duration of the trial

For Idiopathic Participants

  • A diagnosis of idiopathic Parkinson's disease confirmed by the presence of bradykinesia plus one or both of the following symptoms: rigidity or resting tremor; and with an abnormal DaTscan consistent with a dopaminergic deficit
  • Age 40 to 75 years
  • Within 5 years of diagnosis of Parkinson's disease

For Genetic Subtype Participants

  • A confirmed diagnosis of Parkinson's disease plus a genetic diagnosis consistent with Parkinson's disease, specifically PTEN-induced kinase 1 (PINK1), parkin, Leucine-rich repeat kinase 2 (LRRK2) or other mitochondrial genetic subtype
  • Age 21 to 75 years

Exclusion Criteria:

  • Allergy to PTC-589 or other components of the PTC-589 tablet formulation
  • Use of antioxidant supplements, specifically vitamins E and C beyond the recommended daily allowance
  • Other Parkinsonian disorders
  • Montreal Cognitive Assessment (MoCA) score of <24
  • Revised Hamilton Rating Scale for Depression ≥11
  • Parkinsonism due to drugs or toxins
  • Diagnosis of any other clinically significant neurologic disease that will confound the assessment of effect of study drug on disease progression
  • Malignancy within past 2 years
  • Pregnant or plans to become pregnant or breast feeding
  • History of stroke
  • History of brain surgery
  • Hepatic insufficiency with liver function tests (LFTs) >3 times upper limit of normal
  • Renal insufficiency as defined by creatinine >1.5 times normal
  • End stage cardiac failure
  • Participation within past 3 months and for duration of study in a trial of a device, drug, or other therapy for Parkinson's disease

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: PTC589
Participants with Parkinson's disease (idiopathic and mitochondrial genetic subtype participants) will receive PTC589 at a dose of 500 milligrams (mg) (2 tablets of 250 mg each) orally twice daily (BID) for up to 3 months unless discontinued for safety or tolerability issues.
Drug: PTC-589
PTC-589 is a redox active molecule and will be provided in a 250 mg tablet formulation.
Other Names:
  • (R)-Troloxamide quinone

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Participants With Drug-Related Serious Adverse Events (SAEs)
Time Frame: Baseline up to 30 days after last dose of study drug (up to 4 months)
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Baseline up to 30 days after last dose of study drug (up to 4 months)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3
Time Frame: Baseline, Month 3
The MDS-UPDRS is a tool for monitoring the impact of Parkinson's disease, the degree of disability caused, and complications from treatment. Part I (13 items) evaluates nonmotor experiences of daily living (nM-EDL); Part II (13 items) evaluates motor experiences of daily living (M-EDL; Part III (18 items) is a motor examination; Part IV (6 items) examines motor complications (for example, motor fluctuations and dyskinesias). Each item was rated on a 5-point scale, ranging from 0 (normal) to 4 (severe), with higher score indicating greater severity and more impairment. Total score for Part I (nM-EDL) and Part II (M-EDL) each ranges from 0-52; for Part III (motor examination) ranges from 0-72; and for Part IV (motor complications) ranges from 0-24; with higher scores in each range for all 4 parts reflecting greater severity.
Baseline, Month 3
Change From Baseline in Non-motor Symptoms Scale (NMSS) Total Score at Month 3
Time Frame: Baseline, Month 3
Non-motor symptoms were evaluated using the NMSS which was divided into 30 questions in 9 different domains including such symptoms as dribbling saliva, constipation, depression, sleep disorders, apathy, hallucinations and dementia. Symptoms were quantified based on their severity (using a scale of 0 [none] to 3 [severe]) and frequency (using a scale of 0 [rarely] to 4 [very frequent]). Total score derived from adding up the product of the frequency score times severity score for each of the 30 questions. Total score ranged from 0 to 360, with a lower score indicating fewer symptoms.
Baseline, Month 3
Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3
Time Frame: Baseline, Month 3
The PDQ-39 is a self-administered questionnaire for participants with Parkinson's disease that has 39 questions grouped in 8 dimensions: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Each item was scored on a 5-point Likert scale (0 to 4) to indicate the frequency of each event; 0 = never, 1 = occasionally, 2 = sometimes, 3 = often, and 4 = always or cannot do at all. Each dimension's total score ranged from 0-100, with lower scores indicating better health, and higher scores indicating more severe symptoms.
Baseline, Month 3
Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3
Time Frame: Baseline, Month 3
EQ-5D is a questionnaire designed to provide measures of health-related quality of life states, consisting of 5 domains (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension has a 3 point response scale designed to indicate the level of the problem: 1 = no problems, 2 = some problems, 3 = extreme problems. A higher score indicated an increase in the level of problem. The EQ-5D also contains a visual analog scale (EQ-VAS), which records the respondent's self-rated health status on a vertical graduated visual analog scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state). Higher score indicated improvement.
Baseline, Month 3
Montreal Cognitive Assessment (MoCA) Score
Time Frame: Month 3
MoCA is a 30-point questionnaire for cognitive dysfunction. It assesses different cognitive domains: attention and concentration, executive functions, memory, language, visuoconstructional skills, conceptual thinking, calculations, and orientation. Scores on the MoCA range from 0-30 with 26-30 indicating normal global cognition; 18-25 mild cognitive impairment; 10-17 moderate cognitive impairment; and <10 severe cognitive impairment.
Month 3
Beck Depression Inventory (BDI) Score
Time Frame: Month 3
The BDI is a self-reporting 21-item scoring tool that measures characteristic attitudes and symptoms of depression, including physical symptoms. Each of the 21 items on BDI tool represent a depressive symptom. The symptoms are each scored on a 4-point Likert scale of 0 (symptom is absent) to 3 (symptom is severe). Scores for each symptom are added up to obtain the total scores for all 21 items, which are interpreted as follows 1-10 (normal); 11-16 (mild mood disturbance); 17-20 (borderline clinical depression); 21-30 (moderate depression); 31-40 (severe depression); and >40 (extreme depression). Participants with symptom score of 0 were not included in the summary.
Month 3
Change From Baseline in Montgomery and Asberg Depression Rating Scale (MADRS) Total Score at Month 3
Time Frame: Baseline, Month 3
The MADRS is a clinician-rated tool for measuring changes in depressive symptom severity. Ten core symptoms and cognitive features (feelings of sadness, lassitude, pessimism, inner tension, suicidality, reduced sleep or appetite, difficulty concentrating, and a lack of interest) were rated on a severity scale of 0 (no symptoms)) to 6 (symptoms of maximum severity). The total score was the sum of the scores on the 10 items, ranging from 0 to 60 with a higher score indicating increasing depressive symptoms.
Baseline, Month 3
Change From Baseline in Time to Complete Time Up and Go (TUG) Test in ON State at Month 3
Time Frame: Baseline, Month 3
Timed motor tests are simple, objective, quantitative measures for the assessment of Parkinson's disease. They include, in on-medication and off-medication state, timed recorded physical movements. Time Up and Go Test (TUG) is one of timed motor tests which is used to assess a person's mobility and requires both static and dynamic balance. This is a walking assessment. Participants start in the seated position, stand up, walk 7 meters, turn around, and sit back down. The entire process from leaving the chair to returning to the chair was timed. The total time was summarized under ON state with participants on dopamine therapy.
Baseline, Month 3
Maximum Observed Plasma Concentration (Cmax) of PTC589
Time Frame: 0 hour (predose) and 0.5, 1, 2, 4, 6, 8, and 12 hours postdose at Month 1 and 3
0 hour (predose) and 0.5, 1, 2, 4, 6, 8, and 12 hours postdose at Month 1 and 3
Level of Disease-Related Biomarker (Glutathione) in Plasma
Time Frame: Month 3
Glutathione lowest limit of quantification (LLOQ) = 0.01 micromoles (uM) and upper limit of quantification (ULOQ) = 27.83 uM in plasma.
Month 3
Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF)
Time Frame: Month 3
Glutathione LLOQ = 0.002 uM and ULOQ = 0.35 uM in CSF.
Month 3
Level of Disease-Related Biomarker (Glutathione) in Urine
Time Frame: Month 3
Glutathione LLOQ = 0.01 uM, and ULOQ = 1.39 uM in urine.
Month 3

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Edison Pharmaceuticals Inc

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Matthew B Klein, MD FACS, Edison Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 17, 2016

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 8, 2019

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 8, 2019

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 20, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 2, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 4, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 3, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 6, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • EPI589-15-002

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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