A Collection of Case Studies in Infants With Urea Cycle Disorders (UCD) to Evaluate Infant Growth and the Safety of a New Medical Food for UCD
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 3
- 试验地点
- 2
- 主要终点
- Growth: head circumference
研究概览
简要总结
Original study: Study aims to enroll 3-5 children with confirmed Urinary Cycle Disorder (UCD). Subjects meeting the inclusion and exclusion criteria are included during the baseline visit. All subjects will receive the investigational product for a period of 16 weeks. At baseline, 2, 4, 8, 12 and 16 weeks the study parameters are assessed.
The study amendment aims to collect case studies retrospectively of children who have used UCD Anamix Infant for at least 16 weeks, in countries where UCD Anamix Infant is already available on the market. It is aimed to collect the same study parameters of the original study at preferably the same timepoints.
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
- — 至 30 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Prospective study:
- •Male and female infants diagnosed with UCD, aged from birth to 30 days of age
- •Parent / primary caregiver is able to give informed consent
- •Infants who will receive and will be able to take study medical food as part (40-70%) of their protein requirements in the day
- •Retrospective study:
- •Male and female term infants diagnosed with UCD, that started UCD Anamix Infant when aged from birth to 30 days of age.
- •Parent / primary caregiver is able to give informed consent
- •Infants who received UCD Anamix Infant as part of their protein requirements (40-70%) in the day for at least 16 weeks.
排除标准
- •Infants < 5lb 8oz (< 2.5 kg) at birth
- •Infants <34 weeks of gestation and using a special premature formula at study start
- •Infants with severe concurrent illness or major congenital malformations, except conditions consequent to urea cycle disorder
- •Infants with suspected or documented systemic or congenital infections (e.g. human immunodeficiency virus)
- •Infants with evidence of significant cardiac, respiratory, haematological, gastrointestinal, or other systemic diseases
- •Infants currently taking cytotoxic drugs
- •Investigator's uncertainty about the willingness or ability of the parent to comply with the protocol requirements
- •Participation in any other study involving investigational products concomitantly or prior to entry into the study
- •An infant of any personnel connected with the study
- •Infants whose parent / primary caregiver is younger than the legal age of consent
结局指标
主要结局
Growth: head circumference
时间窗: 16 weeks
head circumference \[cm\]
Growth: weight
时间窗: 16 weeks
weight \[kg\]
Growth: length
时间窗: 16 weeks
length \[cm\]
次要结局
- Stool consistency(16 weeks)
- Compliance: product intake(16 weeks)
- GI symptoms [absent/mild/moderate/severe](16 weeks)
- Stool frequency(16 weeks)
- Protein and iron levels from blood samples (for retrospective study part only if data available)(16 weeks)