NCT00718341已完成2 期
A Multi-centre, Randomized, Double-blind, Placebo Controlled, Two-period, Crossover Proof-of-concept Study in Male Patients With Fragile X Syndrome to Assess the Efficacy, Safety and Tolerability of Multiple Oral Doses of AFQ056
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Novartis
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Aberrant-Behavior Checklist- Community Edition
研究概览
简要总结
This study will evaluate the safety, tolerability and efficacy of multiple doses of AFQ056 in patients with Fragile X Syndrome. The dose range will be 50 to 150 mg b.i.d. The primary read-out of efficacy is reduction in Aberrant-Behavior Checklist score.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 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 35 Years（Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male, non-smoking patients between 18 and 35 years of age (both inclusive).
- •Patients with fmr1 full mutation (> 200 CGG repeats)
- •Patients with a Clinical Global Impression Severity Score (CGI-S) of > 4 (moderately ill)
- •Patients with a score of >20 in the ABC-C scale (at screening)
- •Patients with a mental age of ≥ 48 months as measured by the Stanford-Binet test
排除标准
- •Patients with DSM-IV diagnosis of schizophrenia, history and/or presence of psychosis, confusional states and/or repeated hallucinations.
- •Patients with a history of seizures in the past 5 years without any therapeutic treatment controlling the disorders.
- •Patients under stable anti-convulsant therapies that experienced seizures in the 2 years prior to randomization
- •Patients with ECG abnormalities, autonomic dysfunctions, bronchospastic diseases, drug or atopic allergy
- •Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of drugs
- •Patients using (or have used within four weeks before randomization) concomitant medications that are potent inhibitors of CYP3A4 (e.g., ketoconazole, ritonavir, etc.)
- •Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
1
Active Comparator
干预措施: AF056 (Drug)
2
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Aberrant-Behavior Checklist- Community Edition
次要结局
- 28 days treatment with AFQ056 on behavior (communication, socialization, daily living, repetitive behaviors, anxiety/avoidance, clinical global improvement)
- 28 days treatment with AFQ056 on cognition (receptive language, attention, vigilance…)
研究者
研究点 (1)
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