Safety and Biomarker Study of PTC-589 in Participants With Parkinson's Disease
Condition: Parkinson's Disease
MeSH terms: Parkinson Disease
Intervention: PTC-589
Intervention description: PTC-589 is a redox active molecule and will be provided in a 250 mg tablet formulation.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 44.0
Sex: All
Minimum age: 21 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: Parkinson's Disease; PD; EPI-589; EPI589

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

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