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.
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: * Review of medical records. * A brief questionnaire. * Child Behavior Checklist (CBCL) from the Achenbach System of Empirically Based Assessment (for children more than 1.5 years old) * Bayley-IV Social-Emotional Scale assessment (for children less than 1.5 years old)
Study Type
OBSERVATIONAL
Enrollment
158
Data and biospecimens will be collected from two groups of children: * Children who have taken PEG 3350 daily for at least 30 days. * Children who have NOT taken PEG 3350 for at least 30 days. No changes to medicines are recommended as part of this study.
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
PEG 3350 related molecules in plasma
Plasma levels of selected PEG 3350 related molecules will be measured by mass spectrometry using validated assays.
Time frame: 15 minutes
PEG 3350 related molecules in urine
Urine levels of selected PEG 3350 related molecules will be measured by mass spectrometry using validated assays.
Time frame: 10 minutes
PEG 3350 related molecules in medicine samples
Medicine sample levels of selected PEG 3350 related molecules will be measured by mass spectrometry using validated assays.
Time frame: 5 minutes
Neurobehavioral symptoms in children who are taking PEG 3350
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.
Time frame: 5 minutes
Baseline neurobehavioral symptoms in children who are not taking PEG 3350
Participants will complete a short symptom questionnaire specific to this study.
Time frame: 5 minutes
Assessment of current Child behavior (1.5 years to 18 years old)
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%.
Time frame: 30 minutes
Assessment of current Child behavior (< 1.5 years old)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
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.
Time frame: 90 minutes