Polyethylene Glycol Safety in Children

NCT05424757 | Completed FDA Drug

• 2 other identifiers: 14-0114541R01FD005312-01
• Study Type: observational
• Target: 158
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Polyethylene Glycols; Children, Only

Outcome Measures

Primary Outcomes (7)

• PEG 3350 related molecules in plasma

  Plasma levels of selected PEG 3350 related molecules will be measured by mass spectrometry using validated assays.

  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.

  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.

  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.

  5 minutes

• Baseline neurobehavioral symptoms in children who are not taking PEG 3350

  Participants will complete a short symptom questionnaire specific to this study.

  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%.

  30 minutes

• Assessment of current Child behavior (< 1.5 years old)

  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.

  90 minutes

Study Arms (2)

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: 1. Children with no known bowel or nervous system disease or neuropsychiatric symptoms. 2. Children with bowel problems that might increase intestinal permeability. 3. Children with neurologic disease or with neuropsychiatric disorders or symptoms.

Drug: PEG 3350

Children who are not taking PEG 3350

* Children less than 17 years old who have NOT taken PEG 3350 for at least 30 days. Children in three subgroups will be included: 1. Children with no known bowel or nervous system disease or neuropsychiatric symptoms. 2. Children with bowel problems that might increase intestinal permeability. 3. Children with neurologic disease or with neuropsychiatric disorders or symptoms.

Interventions

PEG 3350 DRUG

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 who are taking PEG 3350

Eligibility Criteria

• Age: 0 Years - 16 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Study population will be selected from children cared for at The Children's Hospital of Philadelphia.

You may qualify if:

• Children less than 17 years old who have been taking PEG 3350 for at least one month.
• Dose of PEG 3350 greater than or equal to 0.4 grams/kg/day currently recommended by the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) and European Society for Pediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) guidelines (2). Preferences will be given to children taking at least 17 grams/day.
• Normal Physical and neurological development for age. Weight and height percentiles ≥ 5% and ≤ 95% for age.

You may not qualify if:

• Children with underlying bowel problems that might be expected to increase bowel permeability (are included in group 2). In this group we will specifically exclude children with:
• Diarrhea within the past month. Note that PEG 3350 may cause loose stool and this is often the intended therapeutic effect. Therefore, we will exclude children with presumed infectious diarrhea since this may alter gut permeability but will include children whose loose stool is attributed to PEG 3350. If loose or watery stool began after initiation of PEG 3350 or after an increase in PEG 3350 dose within prior 3 days, children may be enrolled for GROUP 1.
• Children whose stool had a recognized diarrhea causing pathogen within the past 4 months will be excluded since the time required to repair epithelial lining is not clear. Enteric pathogens that would prevent enrollment include Salmonella, Shigella, Campylobacter, Yersinia, E. Coli (enterotoxigenic, enteropathogenic, enteraggregative, enterohemorrhagic), Clostridum difficile, Rotavirus, Norovirus, Astrovirus, and other known or suspected diarrhea causing organisms.
• H. pylori infection since this causes a stomach inflammation. HIV infection since this may alter bowel permeability (17) and predispose to other types of infection Symptoms neurologic disease or dysfunction (since they will be enrolled in Group 3).
• Group 2: Children with problems that might increase intestinal epithelial permeability.
• Children less than 17 years old who have been taking PEG 3350 for at least one month.
• Dose of PEG 3350 greater than or equal to 0.4 grams/kg/day currently recommended by the NASPGHAN and ESPGHAN guidelines (2). Preferences will be given to children taking at 17 grams/day.
• c. Any of the following problems associated with increased bowel permeability:
• Weight ≤ 5% for age. Malnutrition is associated with increased intestinal permeability.
• Children with celiac disease who are untreated or began treatment within the past 3 months.
• Children with inflammatory bowel disease
• Children with Hirschsprung disease. This intestinal motility disorder predisposes to bowel injury called enterocolitis (18-20).
• Children with intestinal motility disorders other than Hirschsprung disease.
• a. Children with neurologic or neuropsychiatric symptoms. These children will be enrolled in Group 3.
• Group 3: Children with underlying neurologic disease or with neuropsychiatric disorders or symptoms.

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• Food and Drug Administration (FDA): collaborator

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

* Blood: 1.5 mL (up to 6 months of age), 2.5 mL (6 to 24 months old), 5 mL (older than 24 months). No more than 3 mL/kg will be collected from any child. * Urine (10 mL) * Stool (10-20 grams for possible future analyses)

MeSH Terms

Interventions

polyethylene glycol 3350

Study Officials

• Robert O Heuckeroth, MD, PhD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: CROSS SECTIONAL
• Target Duration: 1 Day
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 15, 2022
• First Posted: June 21, 2022
• Study Start: September 30, 2022
• Primary Completion: August 9, 2025
• Study Completion: August 9, 2025
• Last Updated: November 4, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)