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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02462603
Recruitment Status : Completed
First Posted : June 4, 2015
Last Update Posted : May 21, 2020
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

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

Condition or disease Intervention/treatment Phase
Parkinson's Disease Drug: EPI-589 Phase 2

Detailed Description:
This is a within-subject, controlled open-label study seeking to determine if EPI-589 can alter the biochemical signature of PD as assessed by peripheral blood and brain imaging biomarkers. Data will also be collected on disease-relevant clinical measures will be collected.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 44 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2A Safety and Biomarker Study of EPI-589 in Mitochondrial Subtype and Idiopathic Parkinson's Disease Subjects
Actual Study Start Date : March 31, 2016
Actual Primary Completion Date : April 16, 2019
Actual Study Completion Date : April 16, 2019

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: EPI-589 500 milligrams (mg) twice daily (bid)
All enrolled participants will receive treatment with EPI-589
Drug: EPI-589
EPI-589 is a redox active molecule and will be provided in a 250 mg tablet formulation.
Other Name: (R)-Troloxamide quinone




Primary Outcome Measures :
  1. Safety as assessed by drug-related adverse events (AEs) [ Time Frame: 3 months ]
    Safety as assessed by drug related adverse events


Secondary Outcome Measures :
  1. Biomarker assessments as measured in cerebrospinal fluid, blood, and urine [ Time Frame: 3 Months ]
  2. Clinical Disease State as assessed by the Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) [ Time Frame: 3 Months ]
  3. Disease morbidity as assessed by composites scales including Non-motor Symptoms Scale (NMSS), The Parkinson's Disease Questionnaire (PDQ-39) and EuroQol Group 5 Dimensions (EQ-5D) [ Time Frame: 3 Months ]
  4. Cognitive function as assessed by composite scales including Montreal Cognitive Assessment (MoCA) and Dementia Rating Scale-2 (DRS-2) scales [ Time Frame: 3 Months ]
  5. Mood as assessed by composite scales including the Beck Depression Inventory (BDI) and Montgomery Asberg Depression Rating (MADRS) scales [ Time Frame: 3 Months ]
  6. To evaluate changes in maximum serum concentration (Cmax) for all treated subjects pre and 3 months post treatment with EPI-589 [ Time Frame: 3 Months ]
  7. Safety as evidenced by development of drug related adverse events after treament with EPI-589 [ Time Frame: 3 Months ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   21 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02462603


Locations
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United States, California
Cedar's Sinai
Los Angeles, California, United States, 90048
University of California, San Francisco
San Francisco, California, United States, 94115
United States, Massachusetts
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States, 02215
Germany
DZNE Site
Tuebingen, Germany, 72074
United Kingdom
University College of London,Dept. of Clinical Neuroscience
London, United Kingdom, NW3 2PF
Sponsors and Collaborators
PTC Therapeutics
Investigators
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Study Director: Matthew B Klein, MD FACS Edison Pharmaceuticals
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Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT02462603    
Other Study ID Numbers: EPI589-15-002
First Posted: June 4, 2015    Key Record Dates
Last Update Posted: May 21, 2020
Last Verified: May 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by PTC Therapeutics:
Parkinson's Disease
PD
EPI-589
EPI589
Additional relevant MeSH terms:
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Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases