Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin.
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 18
- 试验地点
- 7
- 主要终点
- The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years
研究概览
简要总结
The purpose of this study is to verify that pediatric patients, especially those who are not old enough to swallow capsules, accept the taste and palatability of a new suspension.
详细描述
This is an open, non-randomized, non-controlled, multiple-dose study in 18 pediatric patients. The treatment period is three days, and during the study the subjects will rate the taste and palatability of the suspension or (for younger children) their parents will rate the child´s acceptance of the suspension.
The study consists of a screening period, a 3 day treatment period and a 1 week follow-up period.
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
- 1 Month 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with HT-1 currently managed on Orfadin (nitisinone) capsules.
- •Age from 1 month to less than 18 years.
- •Signed informed consent.
排除标准
- •Any medical condition which in the opinion of the investigator makes the subject unsuitable for inclusion.
- •Enrollment in another concurrent clinical study, or intake of an investigational medicinal product (IMP), within one month prior to inclusion in this study.
- •Foreseeable inability to cooperate with given instructions or study procedures.
研究组 & 干预措施
Orfadin suspension
Drug: nitisinone, oral suspension
干预措施: Nitisinone (Drug)
结局指标
主要结局
The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years
时间窗: Day 3
Patients rated the taste of the suspension. The following grading was applied: 5 (very good taste), 4 (good taste), 3 (neither good nor bad taste), 2 (bad taste) and 1 (very bad taste).
The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years
时间窗: Day 3
The parents of patients aged \<5 years rated their child´s acceptability of the suspension. The following grading was applied: 5 (very well), 4 (well), 3 (neither well nor badly), 2 (badly) and 1 (very badly).
次要结局
- The Palatability Scores on Day 1 (Subjects 5 - < 18 Years)(Day 1)
- The Palatability Scores on Day 2 (Subjects 5 - < 18 Years)(Day 2)
- The Palatability Scores on Day 3 (Subjects 5 - < 18 Years)(Day 3)