Kuvan® (Sapropterin) as a Treatment for Autistic Disorder: An Open Label Extension Protocol
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 41
- 试验地点
- 1
- 主要终点
- Clinical Global Impressions Scale
研究概览
简要总结
This is an open-label extension study available only to subjects who completed an earlier double-blind, placebo-controlled study of sapropterin in children with autism.
详细描述
This is an open-label extension study available only to subjects who completed an earlier double-blind, placebo-controlled study of sapropterin in children with autism. During this protocol, all subjects will be receiving brand-name Kuvan 20 mg/kg/day for 16 weeks; subject who complete the first 16 weeks will have the option of continuing on Kuvan at the same dose for up to 90 days after the last subject has completed the first 16 weeks of this protocol. The purpose of the study primarily is to gather additional information on safety and efficacy in this population.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All subjects must have completed earlier trial, CHC 0901 (NCT00850070)
- •Parents must be willing and able to sign informed consent
排除标准
- •Child failed to complete CHC 0901 (NCT00850070)
研究组 & 干预措施
Kuvan®
Kuvan® (sapropterin) will be administered to all subjects at 20 mg/kg/day for 16 weeks.
干预措施: Kuvan® (Drug)
结局指标
主要结局
Clinical Global Impressions Scale
时间窗: 16 weeks
This is a summary judgment made by a trained clinician based on observed and reported behaviors of the child compared to baseline. It is a 7-point scale (1) very much improved, (2) much improved, (3) minimally improved (4) no change, (5) minimally worse, (6) much worse and (7) very much worse. Chi-square analyses were used to assess change in CHI-I scores (by group, post-test)Mixed-effects regression models determined the main effects attributed to differences by group (BH4 and placebo), time (treated as categorical at levels baseline, 8 weeks, and 16 weeks) and the group-by-time interaction. The mixed-effects models accounted for each participant's outcome data at each time point. The mixed-effects regression model is robust to data dependency that occurs with the repeated assessments of individuals over time \& can handle missing data. We used random intercept and trend modeling that accounts for each individual's initial level of symptom severity/functioning and rate of change/time
次要结局
- Parental Global Assessment(Weeks 8 & 16)
- Preschool Language Scale, 4th Edition (PLS-4)(Weeks baseline (week 16 from CHC-0901), 8 and 16. Primary outcome assessment looked at change between baseline (week 16 from CHC-0901 and week 16 of CHC-0902).)
- Connor's Preschool ADHD Questionnaire(Weeks 8 & 16)
- Aberrant Behavior Checklist (ABC)(Weeks baseline (week 16 from CHC-0901), 8 and 16. Primary outcome assessment looked at change between baseline (week 16 from CHC-0901 and week 16 of CHC-0902).)
- Adverse Events Reporting(Cummulative throughout study)
- Vineland Adaptive Behavior Scale, 2nd Edition(Weeks baseline (week 16 from CHC-0901), 8 and 16. Primary outcome assessment looked at change between baseline (week 16 from CHC-0901 and week 16 of CHC-0902).)
- Children's Yale Brown Obsessive Compulsive Scale(Weeks 8 & 16)
研究者
Glen R. Elliott
Chief Psychiatrist and Medical Director
The Children's Health Council