Quantitative Analysis of Selected PEG 3350 Components and Metabolites in Children's Plasma and Urine
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 158
- 试验地点
- 1
- 主要终点
- PEG 3350 related molecules in plasma
研究概览
简要总结
The primary objective is to measure levels of selected PEG 3350 components and metabolites in the blood and urine of children who are already taking PEG 3350 compared to children who are not taking PEG 3350.
Changes to medical therapy are not recommended as part of this study.
详细描述
For subjects taking PEG 3350, blood and urine will be collected 1.5 to 4 hours after subjects take PEG 3350.
For subjects who are not taking PEG 3350, the same PEG 3350 components and metabolites will be measured in blood and urine.
PEG 3350 containing medicines from study subjects will be analyzed for the same PEG 3350 components and metabolites.
A stool specimen will be collected for future studies.
Additional data will be obtained from:
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 0 Years 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Children who are taking PEG 3350
- Children less than 17 years old who have taken PEG 3350 daily for at least 30 days.
- Dose of PEG 3350 greater than or equal to 0.4 grams/kg/day.
- Preference will be given to children taking at least 17 grams/day.
Notes:
Only children who are already taking PEG 3350 as part of their current medical regimen will be included. Changes to medical therapy are not recommended as part of this study.
Children in three subgroups will be enrolled:
- Children with no known bowel or nervous system disease or neuropsychiatric symptoms.
- Children with bowel problems that might increase intestinal permeability.
- Children with neurologic disease or with neuropsychiatric disorders or symptoms.
干预措施: PEG 3350 (Drug)
结局指标
主要结局
PEG 3350 related molecules in plasma
时间窗: 15 minutes
Plasma levels of selected PEG 3350 related molecules will be measured by mass spectrometry using validated assays.
PEG 3350 related molecules in urine
时间窗: 10 minutes
Urine levels of selected PEG 3350 related molecules will be measured by mass spectrometry using validated assays.
PEG 3350 related molecules in medicine samples
时间窗: 5 minutes
Medicine sample levels of selected PEG 3350 related molecules will be measured by mass spectrometry using validated assays.
Baseline neurobehavioral symptoms in children who are not taking PEG 3350
时间窗: 5 minutes
Participants will complete a short symptom questionnaire specific to this study.
Assessment of current Child behavior (1.5 years to 18 years old)
时间窗: 30 minutes
Families with children \>1.5 years to 18 years old will complete an age-appropriate Child Behavior Checklist (CBCL) from the Achenbach System of Empirically Based Assessment. Specific CBCL forms are available for ages 1.5 to 5 years and for 6 to 18 years. Standard scores are scaled. 50 is average for age and gender. Standard deviation is 10 points. Higher scores indicate greater problems. Normal = below 93%. Borderline = 93-97%. Clinical range = above 97%.
Assessment of current Child behavior (< 1.5 years old)
时间窗: 90 minutes
Caregivers with children \< 1.5 years old will complete a Bayley-4 Social-Emotional Scale (BSID-IV) assessment. The BSID-IV Social-Emotional Scale is an adaptation of the Greenspan Social-Emotional Growth Chart: A Screening Questionnaire for Infants and Young Children. The Social-Emotional Scale assesses acquisition of social and emotional milestones. Items assess mastery of functional emotional skills, such as self-regulation and interest in the world; communicating needs; engaging others and establishing relationships; using emotions in a purposeful manner; and using emotional signals to solve problems. The standardized mean motor score is 100 (SD 15), with scores lower than 85 indicating mild impairment, and lower than 70 indicating moderate or severe impairment.
Neurobehavioral symptoms in children who are taking PEG 3350
时间窗: 5 minutes
Participants will complete a short symptom questionnaire specific to this study. The questionnaire asks if specific symptoms are present and if they changed after the child started PEG 3350 containing medicine.
次要结局
未报告次要终点