Analyzing Mycophenolate Mofetil in Children After Stem Cell Transplant

Population Pharmacokinetic and Pharmacodynamic Model-based Dosing Strategy of Mycophenolate Mofetil in Pediatric Hematopoietic Stem Cell Transplantation(HSCT) Patients

Phase 1 Interventional Seoul National University Hospital · NCT04868786

This study is testing how mycophenolate mofetil works in children who have had a stem cell transplant to find the right dose for them.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment20 (estimated)
AgesN/A to 17 Years
SexAll
SponsorSeoul National University Hospital Academic / other
Locations1 site (Seoul)
Trial IDNCT04868786 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the pharmacokinetics and pharmacodynamics of mycophenolate mofetil in pediatric patients who have undergone hematopoietic stem cell transplantation. The study aims to determine the appropriate dosing of this immunosuppressant in children by analyzing its population pharmacokinetic characteristics. Participants must have received mycophenolate treatment for at least three days post-transplant. The trial is led by Seoul National University Hospital and involves collaboration with the National Institute of Food and Drug Safety Evaluation in South Korea.

Who should consider this trial

Good fit: Ideal candidates are children under 18 years who have received mycophenolate mofetil for immunosuppression after allogenic hematopoietic stem cell transplantation.

Not a fit: Patients with known hypersensitivity to mycophenolate mofetil or those in critical medical conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could optimize immunosuppressive therapy in pediatric patients undergoing stem cell transplantation, improving their outcomes.

How similar studies have performed: While this approach is focused on pediatric patients, similar studies in adult populations have shown success in optimizing dosing for mycophenolate mofetil.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Patients who had mycophenolate treatment at least 3 days for immunosuppressant after allogenic hematopoietic stem cell transplantation
* Patients age \<18 years
* Written Study Informed consent and/or assent from the patient, parent, or guardian

Exclusion Criteria:

* Known hypersensitivity to mycophenolate mofetil or similar class of drug substance
* Patients in medically critical condition such as severe infection or unstable vital signs
* Any condition that would, in the Investigator's judgment, interfere with full participation in the study
* Patients with hypoxanthine-guanine phosphoribosyltransferase deficiency(HGPRT) such as Lesch-Nyhan or Kelley-Seegmiller syndrome)

Where this trial is running

Seoul

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.
Conditions Hematopoietic Stem Cell Transplantation
Last reviewed 2026-06-13 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.