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

NCT02462603 | Completed Phase 2 Results FDA Drug

• 1 other identifier: EPI589-15-002
• Study Type: interventional
• Target: 44
• Locations: 3 countries
• Sites: 5

Brief Summary

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

Conditions

Parkinson's Disease

Keywords

Parkinson's Disease; PD; EPI-589; EPI589

Outcome Measures

Primary Outcomes (1)

• 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'.

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

Secondary Outcomes (12)

• Change From Baseline in Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Score at Month 3

  Baseline, Month 3

• Change From Baseline in Non-motor Symptoms Scale (NMSS) Total Score at Month 3

  Baseline, Month 3

• Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3

  Baseline, Month 3

• Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3

  Baseline, Month 3

• Montreal Cognitive Assessment (MoCA) Score

  Month 3

Study Arms (1)

PTC589

EXPERIMENTAL

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.

Drug: PTC-589

Interventions

PTC-589 DRUG

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

Also known as: (R)-Troloxamide quinone

PTC589

Eligibility Criteria

• Age: 21 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Edison Pharmaceuticals Inc: lead

Study Sites (5)

Cedar's Sinai

Los Angeles, California, 90048, United States

Location

University of California, San Francisco

San Francisco, California, 94115, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

DZNE Site

Tübingen, 72074, Germany

Location

University College of London,Dept. of Clinical Neuroscience

London, NW3 2PF, United Kingdom

Location

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Results Point of Contact

• Title: Patient Advocacy
• Organization: PTC Therapeutics, Inc.

medinfo@ptcbio.com

1-866-562-4620

Study Officials

• Matthew B Klein, MD FACS

  Edison Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 20, 2015
• First Posted: June 4, 2015
• Study Start: May 17, 2016
• Primary Completion: January 8, 2019
• Study Completion: January 8, 2019
• Last Updated: May 3, 2022
• Results First Posted: May 3, 2022

Record last verified: 2022-04

Locations

GB (1); DE (1); US (3)