Pharmacokinetics and Pharmacodynamics of Mycophenolate Mofetil in Pediatric Hematopoietic Stem Cell Transplantation

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

This is an investigator-initiated clinical trial to analysis population pharmacokinetic characteristics and investigate appropriate pediatric dose of mycophenolate mofetil in pediatric hematopoietic stem cell transplantation patients.

Study Overview

• Status: Recruiting
• Conditions: Hematopoietic Stem Cell Transplantation
• Intervention / Treatment: Drug: Mycophenolate Mofetil

• Study Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Hyoung Jin Kang, MD, PhD; Phone Number: +82-2-2072-3304; Email: kanghj@snu.ac.kr
• Study Contact Backup: Name: Kyung Taek Hong, MD; Phone Number: +82-2-2072-3631; Email: hongkt@snu.ac.kr

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of, 03080
    • Recruiting
    • Seoul National University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 17 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: PREVENTION
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Mycophenolate Mofetil	Drug: Mycophenolate Mofetil; Administer 15-20mg/kg of mycophenolate mofetil twice daily. Blood sampling for pharmacokinetics(PK) of mycophenolate mofetil will be performed in all patients who have taken the investigational drug for at least 3 days.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetics parameter of melphalan	Analysis: Maximum plasma drug concentration (Cmax)	pre dose, post 1 hour, post 2 hour, post 6 hour
Pharmacokinetics parameter of melphalan	Analysis: Time to reach maximum plasma concentration (tmax)	pre dose, post 1 hour, post 2 hour, post 6 hour
Pharmacokinetics parameter of melphalan	Analysis: Area under the plasma concentration-time curve (AUC)	pre dose, post 1 hour, post 2 hour, post 6 hour
Pharmacokinetics parameter of melphalan	Analysis: Apparent total clearance of the drug from plasma after oral administration (CL/F)	pre dose, post 1 hour, post 2 hour, post 6 hour
Pharmacokinetics parameter of melphalan	Analysis: Apparent volume of distribution after non-intravenous administration (V/F)	pre dose, post 1 hour, post 2 hour, post 6 hour

Collaborators and Investigators

• Sponsor: Seoul National University Hospital
• Collaborators: National Institute of Food and Drug Safety Evaluation (Republic of Korea)
• Investigators: Principal Investigator: Hyoung Jin Kang, MD, PhD, Seoul National University Hospital

Study record dates

Study Major Dates

• Study Start (ACTUAL): October 26, 2020
• Primary Completion (ANTICIPATED): July 1, 2025
• Study Completion (ANTICIPATED): October 1, 2025

Study Registration Dates

• First Submitted: March 28, 2021
• First Submitted That Met QC Criteria: April 29, 2021
• First Posted (ACTUAL): May 3, 2021

Study Record Updates

• Last Update Posted (ACTUAL): February 6, 2023
• Last Update Submitted That Met QC Criteria: February 3, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Enzyme Inhibitors; Antineoplastic Agents; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antitubercular Agents; Antibiotics, Antitubercular; Mycophenolic Acid
• Other Study ID Numbers: SNUCH-MIDD-MMF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes