Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008: Cognition in Participants With Pyruvate Kinase Deficiency
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 13
- 试验地点
- 3
- 主要终点
- Change Over Time in Cogstate Brief Battery (CBB) Scores
研究概览
简要总结
This is a substudy of Study AG348-C-008, an observational, longitudinal, multicenter, global registry that aims to better understand the longitudinal clinical implications of pyruvate kinase deficiency (PK deficiency). The primary purpose of this study is to describe the cognition of participants with PK deficiency, as objectively measured by the Cogstate Brief Battery (CBB).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be ≥18 years of age;
- •Participant must be enrolled in Study AG348-C-008.
排除标准
- 未提供
结局指标
主要结局
Change Over Time in Cogstate Brief Battery (CBB) Scores
时间窗: Day 0, Day 90
CBB is a series of brief computerized assessments used to evaluate 4 major cognitive domains: psychomotor speed, attention/vigilance, visual learning, and working memory. If the CBB score falls between -10 to +10, it is considered normal. In Cogstate scoring 0 could be considered as performing in the average range, a negative score means below-average performance, and a positive score means above-average performance. A positive change in scores indicates slightly better performance.
次要结局
- CBB Scores of Participants as Compared to the Scores of Age-Matched Controls(Day 0, Day 90)
- Percentage of Participants With Blood Transfusions Affecting Cognition, as Assessed by the Investigator(Day 90)