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

Edison Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Parkinson's Disease

Treatments: Drug: PTC-589

Study type: Interventional

Funder types: Industry

Identifiers

NCT02462603

EPI589-15-002

Details and patient eligibility

About

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

Full 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.

Enrollment: 44 patients

Sex: All

Ages: 21 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

44 participants in 1 patient group

PTC589

Experimental group

Description:

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.

Treatment:

Drug: PTC-589