A Phase 2A Safety and Biomarker Study of EPI-589 in Mitochondrial Subtype and Idiopathic Parkinson's Disease Subjects
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 44
- 试验地点
- 5
- 主要终点
- Number of Participants With Drug-Related Serious Adverse Events (SAEs)
研究概览
简要总结
Open-label study with 30-day run-in phase and adaptive design component to include more participants if deemed appropriate by investigators.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 21 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
PTC589
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.
干预措施: PTC-589 (Drug)
结局指标
主要结局
Number of Participants With Drug-Related Serious Adverse Events (SAEs)
时间窗: Baseline up to 30 days after last dose of study drug (up to 4 months)
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'.
次要结局
- Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF)(Month 3)
- Change From Baseline in Parkinson's Disease Questionnaire - 39 (PDQ-39) Score at Month 3(Baseline, Month 3)
- Change From Baseline in Time to Complete Time Up and Go (TUG) Test in ON State at Month 3(Baseline, Month 3)
- Maximum Observed Plasma Concentration (Cmax) of PTC589(0 hour (predose) and 0.5, 1, 2, 4, 6, 8, and 12 hours postdose at Month 1 and 3)
- Level of Disease-Related Biomarker (Glutathione) in Urine(Month 3)
- Change From Baseline in Non-motor Symptoms Scale (NMSS) Total Score at Month 3(Baseline, Month 3)
- Change From Baseline in EuroQol-5 Dimension (EQ-5D) Score at Month 3(Baseline, Month 3)
- Beck Depression Inventory (BDI) Score(Month 3)
- Change From Baseline in Montgomery and Asberg Depression Rating Scale (MADRS) Total Score at Month 3(Baseline, Month 3)
- Level of Disease-Related Biomarker (Glutathione) in Plasma(Month 3)
- 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)
- Montreal Cognitive Assessment (MoCA) Score(Month 3)