Safety and Biomarker Study of EPI-589 in Parkinson's Disease

• Status: Completed
• Trial end date: 2019-04-16
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Edison Pharmaceuticals Inc; PTC Therapeutics

Criteria

Inclusion Criteria:

• Hoehn and Yahr stage ≤ 3.0

• Ambulatory with or without assistance

• Sexually active fertile subjects 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 PD drugs, then medication regimen must be stable for 60 days prior to enrollment

• Abstention from use of other investigative or nonapproved drugs for the duration of the trial.

For Idiopathic Subjects

• A diagnosis of idiopathic PD 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 Subjects

• A confirmed diagnosis of PD plus a genetic diagnosis consistent with PD, specifically PINK1, parkin, LRRK2 or other mitochondrial genetic subtype

• Age 21 to 75 years

Exclusion Criteria:

• Allergy to EPI-589 or other components of the EPI-589 tablet formulation

• Use of antioxidant supplements, specifically vitamins E and C beyond the recommended daily allowance

• Other Parkinsonian disorders

• 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 two 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 PD