Measuring how off-label medications affect children

Pharmacokinetics of Off Label Pediatric Medications

University of North Carolina, Chapel Hill · NCT01286519

This study is testing how off-label medications affect young children by looking at drug levels in their blood and other fluids to better understand their safety and effectiveness.

Quick facts

Study typeObservational
Enrollment400 (estimated)
Ages1 Day to 8 Years
SexAll
SponsorUniversity of North Carolina, Chapel Hill (other)
Locations1 site (Chapel Hill, North Carolina)
Trial IDNCT01286519 on ClinicalTrials.gov

What this trial studies

This study aims to assess the pharmacokinetics of medications used off-label in pediatric patients by analyzing levels of these drugs in body fluids such as blood, urine, and spinal fluid. It addresses the significant gap in safety and efficacy data for therapeutic agents in children under 8 years of age. The study utilizes scavenged samples, which are extra fluids collected during routine medical tests, to gather necessary data without additional procedures. This observational approach allows for the collection of real-world data on medication effects in a vulnerable population.

Who should consider this trial

Good fit: Ideal candidates for this study are children under 8 years old who are receiving medications not currently approved by the FDA for their specific use.

Not a fit: Patients who do not provide consent will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could enhance the understanding of how off-label medications work in children, leading to safer and more effective treatments.

How similar studies have performed: While there is limited data on similar studies, the approach of using scavenged samples for pharmacokinetic analysis in pediatric populations is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Children less than 8 years of age
* Receiving a therapeutic agent at a dose or for an indication not currently included on the FDA label as part of standard of care

Exclusion Criteria:

* Lack of consent

Where this trial is running

Chapel Hill, North Carolina

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Off Label Use of Medications in Pediatric Patients, pharmacokinetics

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.