A Randomized, Double-Blind, Placebo-Controlled, Dose Escalation Study of Single and Multiple Oral Dose Administration of BIIB014 in Subjects With Moderate to Late Stage Parkinson's Disease Who Are Also Receiving Treatment With Levodopa
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Biogen
- 入组人数
- 83
- 试验地点
- 2
- 主要终点
- Number and proportion of subjects with adverse events
研究概览
简要总结
The main purpose of this study is to determine the safety of BIIB014 and how well BIIB014 is tolerated when given at different doses to patients with moderate to late-stage Parkinson's Disease who are also taking the Parkinson's medication, levodopa (L-DOPA).
This study will also explore:
- the pharmacokinetics of BIIB014 in Parkinson's patients who are also taking L-DOPA (this will be done by measuring the levels of BIIB014 in the blood at several different times during the study), and
- the activity of BIIB014 when given to Parkinson's patients who are also taking L-DOPA (this will be done by performing different Parkinson's Disease assessments during the study to examine change in waking OFF time, change in time with troublesome dyskinesia, change in Unified PD Rating Scale (UPDRS) scores, and Clinical Global Improvement).
Patients who enter this study will be randomly assigned to receive either BIIB014 or a placebo but because the study is blinded, neither they nor their study doctor will know which study treatment they are taking.
The study will be divided into 2 parts:
- Part A: a, rapid, sequential cohort, dose escalation to establish MTD, followed by
- Part B: a parallel-group exploration of the two highest tolerated doses versus placebo.
Note: As Part A of the study is now concluded, some of the study design information presented below (e.g., number of study arms) pertains only to Part B.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 30 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must carry a diagnosis of idiopathic PD according to UK Parkinson's Disease Society Brain Bank Clinical Diagnostic Criteria, and be Hoehn & Yahr Stage II to IV (inclusive) when OFF.
- •Must be on a stable dose of L-3,4-dihydroxyphenylalanine (L-DOPA)/carbidopa or L-DOPA/benserazide for at least 4 weeks prior to enrollment.
- •Except for L-DOPA and certain allowed dopamine agonists, must not be receiving any other PD medications. (Current treatment with certain dopamine agonists is allowed but subjects must have been on a stable dose for at least 4 weeks prior to enrollment).
- •Some subjects must demonstrate a definite end of L-DOPA dose wearing off (at least 2 hours OFF time per waking day) and must be able to keep accurate patient diaries of PD activity.
排除标准
- •A Mini Mental State Examination (MMSE) score <
- •History or clinical features consistent with an atypical parkinsonian syndrome.
- •Any significant non-Parkinson's central nervous system disorder.
- •Any significant AXIS I psychiatric disease from the Diagnostic and Statistical Manual of Mental Disorders (DSM).
- •Any previous surgical intervention for Parkinson's Disease.
- •History of certain malignancies.
- •History of severe allergic anaphylactic reactions to any drug.
- •Clinically significant baseline electrocardiogram (ECG).
- •Orthostatic hypotension.
- •HbA1c >7.0%
研究组 & 干预措施
1
BIIB014 at MTD from Part A
干预措施: BIIB014 (Drug)
2
BIIB014 at dose immediately below MTD from Part A
干预措施: BIIB014 (Drug)
3
干预措施: Placebo (Drug)
结局指标
主要结局
Number and proportion of subjects with adverse events
时间窗: up to end of study
Assessment of clinical laboratory parameters
时间窗: up to end of study
Assessment of vital signs
时间窗: up to end of study
Assessment of ECG parameters.
时间窗: up to end of study
次要结局
- Assess PK by measuring concentrations of BIIB014 and its N-acetyl metabolite in blood plasma.(up to 24h following last dose (Part A only))
- Explore activity of BIIB014 by evaluating standard Parkinson's disease assessments(up to 8h following last dose (Part A); up to 24h following last dose (Part B only))