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

Edison Pharmaceuticals Inc44 enrolled

Overview

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

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

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Parkinson's Disease

Interventions

PTC-589DRUG

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

Eligibility

Sex: ALLMin age: 21 YearsMax age: 75 Years

Medical Language ↔ Plain English

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

Locations (5)

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

Outcomes

Primary Outcomes

Number of Participants With Drug-Related Serious Adverse Events (SAEs)

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'.

Time frame: Baseline up to 30 days after last dose of study drug (up to 4 months)

Secondary Outcomes

Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at 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.

Time frame: Baseline, Month 3

Change From Baseline in Non-motor Symptoms Scale (NMSS) Total Score at 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.

Data from ClinicalTrials.gov

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