NCT01750957已完成2 期
A Randomized, Parallel Group, Double-Blind, Placebo-Controlled, Safety and Exploratory Efficacy and Pharmacokinetic, Study of RO4917523 in Pediatric Patients With Fragile X Syndrome
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 47
- 主要终点
- Safety: Incidence of adverse events
研究概览
简要总结
This randomized, double-blind, placebo-controlled, parallel-arm study will evaluate the safety and exploratory efficacy and pharmacokinetics of RO4917523 in pediatric patients with fragile X syndrome. Patients will be randomized to receive one of 2 dose levels of RO4917523 or placebo orally daily for 12 weeks.
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
- 5 Years 至 13 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children and adolescents, 5 to 13 years of age
- •Diagnosis of fragile X syndrome based on prior DNA testing confirming Fragile X Mental Retardation 1 (FMR1) full mutation and qualifying scores on the ABC and CGI-S
排除标准
- •Previous treatment with another mGlu5 receptor antagonist within the prior 3 months
- •Participation in a clinical trial involving an investigational drug (unapproved) or non-drug treatment within the prior 6 weeks or 5 times the half-life (whichever is longer) before the start of this study
- •Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome
- •History of suicidal behavior
- •Other protocol defined inclusion/exclusion criteria may apply
研究组 & 干预措施
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
RO4917523 Dose A
Experimental
干预措施: RO4917523 (Drug)
RO4917523 Dose B
Experimental
干预措施: RO4917523 (Drug)
结局指标
主要结局
Safety: Incidence of adverse events
时间窗: 15 weeks
次要结局
- Efficacy: Neuropsychological/behavioral assessment scales (ADAMS/Clinical Global Impressions CGI-S, CGI-I/ GBAS/Aberrant Behavior Checklist ABC/Repeatable Battery for the Assessment of Neuropsychological Status RBANS/VAS behavior)(15 weeks)
- Pharmacokinetics: Clearance (CL/F)(up to Week 12)
- Pharmacokinetics: Volume of distribution at steady-state (Vss/F)(up to Week 12)
研究者
相似试验
已完成
2 期
MARIGOLD Study: A Study of RO4917523 Versus Placebo as Adjunctive Therapy in Patients With Major Depressive Disorder and an Inadequate Response to Ongoing Antidepressant TherapyMajor Depressive DisorderNCT01437657Hoffmann-La Roche319
已完成
2 期
A Study of RO5217790 in Participants With High Grade Cervical Intraepithelial Neoplasia (CIN) Associated With High Risk Human Papillomavirus (HR-HPV) InfectionCervical Intraepithelial NeoplasiaNCT01022346Hoffmann-La Roche206
已完成
1 期
A Study of Aleglitazar in Combination With Aspirin in Healthy VolunteersHealthy VolunteerNCT01188304Hoffmann-La Roche44
已完成
1 期
A Study of Aleglitazar in Combination With Ibuprofen in Healthy VolunteersHealthy VolunteerNCT01188317Hoffmann-La Roche44
已完成
3 期
A Study of Taspoglutide in Patients With Inadequately Controlled Diabetes Mellitus Type 2 and Cardiovascular DiseaseDiabetes Mellitus Type 2NCT01018173Hoffmann-La Roche2,118