NCT03436953撤回2 期
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of CX-8998 for Tremor Associated With Parkinson's Disease
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 主要终点
- Change from Baseline to Day 28 on the MDS-UPDRS Tremor Score as scored by the central rater
研究概览
简要总结
This is a Phase 2, multicenter, double-blind, placebo-controlled, parallel-group study consisting of a screening period of up to 4 weeks, a 4 week randomized double-blind, dose-titration treatment period, followed by a 1 week safety follow-up period after the last dose of study medication, and a scheduled follow-up safety telephone call one week later.
详细描述
This is a Phase 2, multicenter, double-blind, placebo-controlled, parallel-group study consisting of a screening period of up to 4 weeks, a 4 week randomized double-blind, dose-titration treatment period, followed by a 1 week safety follow-up period after the last dose of study medication, and a scheduled follow-up safety telephone call one week later.
Subjects will be randomized 1:1 to one of two treatment groups. Group A will receive titrating doses of CX-8998 up to 10 mg BID and Group B will receive placebo.
Subjects will participate for a total of up to 12 weeks, including screening, the 4-week treatment period and follow-up.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 40 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Men or non-pregnant, non-breastfeeding women 40 to 80 years-of-age who are able to read and understand English.
- •Mini Mental State Exam (MMSE) score ≥
- •Clinical diagnosis of idiopathic Parkinson's disease and presence of at least 2 out of 3 cardinal characteristics (tremor, rigidity, and/or bradykinesia).
- •Hoehn & Yahr Stage I III (inclusive) if not experiencing motor fluctuations. If experiencing motor fluctuations, must be Hoehn & Yahr Stage I IV (inclusive) when OFF or I-III (inclusive) when ON.
- •An MDS-UPDRS tremor score (sum of items 2.10, 3.15, 3.16, 3.17, 3.18) of a least 10 (during ON for subjects experiencing fluctuations) (centrally rated) (Forjaz et al., 2015). A limited number of subjects with an MDS-UPDRS of 8 or 9 may be included with Sponsor approval.
- •Treated with a stable regimen of anti-parkinsonian and/or anti-tremor medication (with the exception of primidone) for at least 2 weeks prior to screening. Changes to anti-parkinsonian or anti-tremor medications after screening is not permitted.
排除标准
- •Current diagnosis of: a. essential tremor / b. cerebellar disease
- •Presence or known history of: a. significant visual hallucinations (in the opinion of the Investigator and/or Study Safety Representative) / b. significant impulse control disorder (ICD) (in the opinion of the Investigator and/or Study Safety Representative).
- •History or clinical features consistent with an atypical parkinsonian syndrome.
- •Dyskinesia or dystonia that would, in the opinion of the investigator, central rater, or Sponsor, interfere with the assessment of tremor.
- •Exposure to tremorigenic drugs or drug withdrawal states within the 30 days prior to the first planned dose of study drug.
- •Direct or indirect trauma to the nervous system within 3 months preceding the onset of tremor.
- •History or clinical evidence of psychogenic tremor origin. Known history of other medical or neurological conditions that may cause or explain subject's tremor.
- •Prior MR-guided Focused Ultrasound or surgical intervention (e.g., deep brain stimulation, ablative thalamotomy or gamma knife thalamotomy) for treatment of tremor or Parkinson's disease.
- •Use of medication(s) in the past month that might produce tremor or interfere with the evaluation of tremor.
- •Inability to refrain from use of medication/substance(s) that might produce tremor or interfere with the evaluation of tremor on study visit days.
- •Positive urine drug screen for drugs of abuse, except if this is explained by use of an allowed prescription medicine.
- •Regular use of more than two units of alcohol per day.
- •Use of prescription or non-prescription drugs or other products (i.e. grapefruit juice) known to be strong inhibitors or inducers of CYP3A4 which cannot be discontinued 2 weeks prior to Day 1 of dosing and withheld throughout the study.
- •Concurrent illnesses that would be a contraindication to trial participation.
- •Psychological, social, familial, or geographical reasons that would hinder or prevent compliance with the requirements of the protocol or compromise the informed consent process.
- •Any other condition and/or situation that causes the Investigator or Study Safety Representative to deem a subject unsuitable for the study (e.g., due to expected study medication non-compliance, inability to medically tolerate the study procedures, or a subject's unwillingness to comply with study-related procedures).
- •Treatment with an investigational agent within 30 days prior to the first dose of CX-8998 or planning to receive an investigational agent during the study.
研究组 & 干预措施
CX-8998 T-type calcium channel blocker
Experimental
干预措施: CX-8998 (Drug)
Comparator
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Change from Baseline to Day 28 on the MDS-UPDRS Tremor Score as scored by the central rater
时间窗: Baseline through completion of study treatment period, an average of 28 days
The MDS-UPDRS is a multi-dimensional scale that assesses the motor and non-motor impact of PD across four parts. Part I: Non-Motor Experiences of Daily Living; Part II: Motor Experiences of Daily Living; Part III: Motor Examination; and Part IV: Motor Complications.
次要结局
- Change from Baseline to Day 28 on the TETRAS Activity of Daily Living subscale(Baseline through completion of study treatment period, an average of 28 days)
- Percentage of subjects who did not complete the study due to Treatment Emergent Adverse Events as assessed by CTCAE v4.0(Duration of study, an average of 12 weeks)
- Columbia-Suicide Severity Rating Scale (C-SSRS)(Screening through study completion, an average of 8 weeks)
- Questionnaire for Impulsive-Compulsive Disorders in Parkinson's Disease-Rating Scale (QUIP-RS)(Screening through study completion, an average of 8 weeks)
- Change from Baseline to Day 28 in accelerometry score(Baseline through completion of study treatment period, an average of 28 days)
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] as assessed by CTCAE v4.0(Through study completion, an average of 12 weeks)
- Changes from baseline in QTcF(Baseline through study completion, an average of 5 weeks)
- Epworth Sleepiness Scale(Baseline through completion of study treatment period, an average of 28 days)
- Percentage of subjects with Serious Adverse Events as assessed by CTCAE v4.0(Duration of study, an average of 12 weeks)
- Percentage of subjects with Adverse Events of Special Interest as assessed by CTCAE v4.0(Duration of study, an average of 12 weeks)
- University of Miami Parkinson's disease Hallucinations Questionnaire (UM-PDHQ)(Screening, Baseline and Day 28, an average of 28 days)
- Hospital Anxiety and Depression Scale (HADS)(Screening and Day 28, an average of 28 days)
研究者
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