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

NCT ID: NCT02462603

Last Updated: 2022-05-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 44 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-05-17
• Study Completion Date: 2019-01-08

Brief Summary

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

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.

Conditions

Parkinson's Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

PTC-589

Intervention Type: DRUG

PTC-589 is a redox active molecule and will be provided in a 250 mg tablet formulation.

Interventions

PTC-589

PTC-589 is a redox active molecule and will be provided in a 250 mg tablet formulation.

Intervention Type: DRUG

Other Intervention Names

(R)-Troloxamide quinone

Eligibility Criteria

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

• Minimum Eligible Age: 21 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Edison Pharmaceuticals Inc

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Matthew B Klein, MD FACS

Role: STUDY_DIRECTOR

Edison Pharmaceuticals

Locations

Cedar's Sinai

Los Angeles, California, United States

University of California, San Francisco

San Francisco, California, United States

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

DZNE Site

Tübingen, , Germany

University College of London,Dept. of Clinical Neuroscience

London, , United Kingdom

Countries

United States; Germany; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

EPI589-15-002

Identifier Type: -

Identifier Source: org_study_id