A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 175
- 试验地点
- 1
- 主要终点
- Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (ABC-C) Total score in Stratum I
研究概览
简要总结
This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective in treating the behavioral symptoms of Fragile X Syndrome.
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
- 18 Years 至 45 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with Fragile X Syndrome, who are at least moderately ill based on a Clinical Global Impression Severity score of at least 4 and have qualifying scores on the ABC-C and IQ test at Visit 1
排除标准
- •Advanced, severe or unstable disease that may interfere with the study outcome evaluations
- •Cancer within the past 5 years, other than localized skin cancer
- •Current treatment with more than two psychoactive medications, excluding anti-epileptics
- •History of severe self-injurious behavior
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
25 mg bid AFQ056
1 capsule of 25 mg and 1 capsule of placebo per intake
干预措施: AFQ056 (Drug)
50 mg bid AFQ056
2 capsules of 25 mg per intake
干预措施: AFQ056 (Drug)
100 mg bid AFQ056
1 capsule of 100 mg and 1 capsule of placebo per intake
干预措施: AFQ056 (Drug)
Placebo
2 capsules of placebo per intake
干预措施: Placebo (Drug)
结局指标
主要结局
Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (ABC-C) Total score in Stratum I
时间窗: 12 weeks
The ABC-C is a 58-item questionnaire that should have been completed as much as possible by the same rater. It is comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech) plus the total score which ranks from 0 to 174 in patients who were fully methylated (FM)
次要结局
- The proportion of patients with clinical response in the ABC-C total score(12 weeks)
- Change from baseline in behavioral symptoms of Fragile X Syndrome (FXS) using the ABC-C Total score in Stratum II(12 weeks)
- Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-C scale(12 weeks)
- Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale(12 weeks)
- improvement of repetitive behavior as measured by changes in the RBS-R(Week 12)