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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02462603
• Date of registration: 20/05/2015
• Prospective Registration: Yes
• Primary sponsor: PTC Therapeutics
• Public title: Safety and Biomarker Study of EPI-589 in Parkinson's Disease
• Scientific title: A Phase 2A Safety and Biomarker Study of EPI-589 in Mitochondrial Subtype and Idiopathic Parkinson's Disease Subjects
• Date of first enrolment: March 31, 2016
• Target sample size: 44
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02462603
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Germany, United Kingdom, United States

Contacts

• Name: Matthew B Klein, MD FACS
• Affiliation: Edison Pharmaceuticals

Key inclusion & exclusion 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



Age minimum: 21 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Parkinson's Disease

Intervention(s)

Drug: EPI-589

Primary Outcome(s)

Safety as assessed by drug-related adverse events (AEs) [Time Frame: 3 months]

Secondary Outcome(s)

Mood as assessed by composite scales including the Beck Depression Inventory (BDI) and Montgomery Asberg Depression Rating (MADRS) scales [Time Frame: 3 Months]

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]

Biomarker assessments as measured in cerebrospinal fluid, blood, and urine [Time Frame: 3 Months]

Safety as evidenced by development of drug related adverse events after treament with EPI-589 [Time Frame: 3 Months]

Cognitive function as assessed by composite scales including Montreal Cognitive Assessment (MoCA) and Dementia Rating Scale-2 (DRS-2) scales [Time Frame: 3 Months]

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]

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]

Secondary ID(s)

EPI589-15-002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors