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

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.

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.

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