A Phase 2, Randomized, Placebo Controlled, Double Blind Proof-of-concept Study Of The Efficacy And Safety Of Pf-02545920 In Subjects With Huntington's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 272
- 试验地点
- 56
- 主要终点
- Change From Baseline in the Total Motor Score (TMS) Assessment of the Unified Huntington Disease Rating Scale (UHDRS) After 26 Weeks of Treatment.
研究概览
简要总结
This study is a 26 week, randomized, parallel group, double blind comparison of PF-02545920 5 mg, PF-02545920 20 mg, and placebo dosed BID in the treatment of motor impairment of subjects with Huntington's Disease. A total of approximately 260 subjects are planned to be randomized in the study. Primary endpoint is the change from baseline in the Total Motor Score (TMS) assessment of the Unified Huntington Disease Rating Scale (UHDRS) after 26 weeks of treatment. secondary endpoints will include change from baseline in the Total Maximum Chorea (TMC) score of the UHDRS after 13 and 26 weeks of treatment and Clinical Global Impression-Improvement score after 13 and 26 weeks of treatment.
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 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •CAG repeat equal or greater than 36;
- •Total motor score equal or greater than 10;
- •Total functional capacity equal or greater than 7.
排除标准
- •Clinically significant neurologic disorder other than Huntington's disease;
- •Other severe acute psychiatric conditions, mania and/or psychosis;
- •History of neutropenia, and myeloproliferative disorders;
研究组 & 干预措施
20 mg PF-02545920 BID
20 mg PF-02545920 BID
干预措施: PF-02545920 (Drug)
5 mg PF-02545920 BID
5 mg PF-02545920 BID
干预措施: PF-02545920 (Drug)
Placebo BID
Matching placebo
干预措施: Placebo (Other)
结局指标
主要结局
Change From Baseline in the Total Motor Score (TMS) Assessment of the Unified Huntington Disease Rating Scale (UHDRS) After 26 Weeks of Treatment.
时间窗: Baseline, Week 26
The UHDRS was a clinical rating scale which has been developed by the Huntington Disease Study Group (HSG) to provide a uniform assessment of the clinical features and course of Huntington's Disease (HD). The components of the full UHDRS assess motor function, cognition, behavior and functional abilities. The total motor score (TMS) assessed motor features of HD with standardized ratings of oculomotor function, dysarthria, chorea, dystonia, gait, and postural stability. Some items (such as chorea and dystonia) required grading each extremity (face, bucco-oral-lingual, and trunk) separately. Eye movements require both horizontal and vertical grades. The total motor impairment scores was the sum of all the individual 31 motor sub-items (each rated from 0 to 4), with higher scores indicating more severe motor impairment than lower scores. The range of TMS is 0-124.
次要结局
- Number of Participants That Met White Blood Count (WBC) and Absolute Neutrophil Count (ANC) Stopping Criteria(Screening, Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192))
- Number of Participants With Adverse Events(Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192))
- Number of Participants With Serious Adverse Events(Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192))
- Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormalities)(Screening, Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192))
- Number of Participants With Laboratory Test Abnormalities (With Normal Baseline)(Screening, Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192))
- Number of Participants With Vital Sign Data That Met Criteria for Potential Clinical Concern (Absolute Values)(Screening, Day 1, 28, 91, and 182)
- Number of Participants With Vital Sign Data That Met Criteria for Potential Clinical Concern (Increase From Baseline)(Screening, Day 1, 28, 91, and 182)
- Number of Participants With Vital Sign Data That Met Criteria for Potential Clinical Concern (Decrease From Baseline)(Screening, Day 1, 28, 91, and 182)
- Number of Participants With Electrocardiogram (ECG) Data That Met Criteria for Potential Clinical Concern(Absolute Values)(Screening, Day 1, 28, 91, and 182)
- Number of Participants With Electrocardiogram (ECG) Data That Met Criteria for Potential Clinical Concern(Increase From Baseline)(Screening, Day 1, 28, 91, and 182)
- Severity of Adverse Events Related to Extrapyramidal Symptoms (EPS) Including Dystonia and Akathisia(Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192))
- Change From Baseline in the Total Maximum Chorea (TMC) Score of the UHDRS After 13 and 26 Weeks of Treatment.(Baseline, Week 13, Week 26)
- Number of Participants With Suicidal Ideation or Suicidal Behavior as Assessed by the Columbia Suicide Severity Rating Scale (C-SSRS) From Baseline to Follow-up Visit(Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192))
- Clinical Global Impression of Improvement (CGI-I) Scale Score After 13 and 26 Weeks of Treatment.(Week 13 & Week 26)