Target Busulfan Exposure in Children With HSCT in China

March 8, 2021 updated by: The First Affiliated Hospital of Soochow University

The Therapeutic Window of Busulfan in Children With Haemopoietic Stem Cell Transplantation：A Multicenter Study in China

Objectives To evaluate the correlation between BU exposure and post-transplant clinical results (efficacy and safety) to establish the optimal BU treatment window for myeloablative conditioning in Chinese pediatrics, provide theoretical basis and the new strategy for BU individualized dosage, further optimize transplant treatment and reduce drug-related toxicity.

Population 500 participants of any sex between the age of 0.1 and 18 years. Patients receiving the BU-based myeloablative conditioning before transplantation.

Endpoint primary To establish BU pop-PK model and analyze the association between BU AUC and event-free survival (EFS)or overall survival (OS) after transplantation in Chinese pediatrics.

Secondary The investigators are also interested in transplantation-related mortality (TRM), acute toxicity and chronic GvHD.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Objectives To evaluate the influence factors of BU metabolism in children and the correlation between BU exposure and post-transplant clinical results (efficacy and safety) to establish the optimal BU treatment window for myeloablative conditioning in Chinese pediatrics, provide theoretical basis and the new strategy for BU individualized dosage, further optimize transplant treatment and reduce drug-related toxicity.

Endpoint primary The main study endpoints are event-free survival (EFS) and overall survival (OS). EFS is calculated from the time of transplant until death, relapse of disease, or graft failure (defined as non-engraftment or rejection), whichever occurred first. OS is the time between transplantation and death of any cause. All surviving patients are censored at day of last contact. Duration of follow-up is the time from transplant to the last assessment for surviving patients or death.

Secondary The investigators are also interested in transplantation-related mortality (TRM), acute toxicity and chronic GvHD. TRM is defined as death unrelated to underlying disease. Acute toxicities are defined as VOD (diagnosed according to modified Seattle criteria), acute GvHD grade II-IV (diagnosed and graded according to Mount Sinai Acute GvHD International Consortium (MAGIC) criteria, oral mucositis (grade II-IV) and hemorrhagic cystitis (grade II-IV). Two of the most commonly utilised scales for oral mucositis are the WHO and NCI-CTCAE scales. Chronic GvHD should be graded as mild, moderate or severe according to the National Institutes of Health (NIH) consensus criteria.

Study Type

Observational

Enrollment (Anticipated)

500

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

  • Name: Xiaohuan Du, MS
  • Phone Number: 86-512-80692268
  • Email: dxhszet@163.com

Study Locations

  • China
    • Jiangsu
      • Suzhou, Jiangsu, China, 215006
        • Recruiting
        • the First Affiliated Hospital of Soochow University
        • Sub-Investigator:
          • Hua Jiang, MD
        • Contact:
        • Contact:
          • Xiaohuan Du, MS
          • Phone Number: 8651280692268 8651280692268
          • Email: dxhszet@163.com
        • Principal Investigator:
          • Liyan Miao, phD
        • Principal Investigator:
          • Shaoyan Hu, MD
        • Sub-Investigator:
          • Xiaowen Qian, MD
        • Sub-Investigator:
          • Yiping Zhu, MD
        • Sub-Investigator:
          • Maoquan Qin, MD
        • Sub-Investigator:
          • Sixi Liu, MD
        • Sub-Investigator:
          • Dao Wang, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 second to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

500 participants of any sex between the age of 0.1 and 18 years. Patients receiving the BU-based myeloablative conditioning before transplantation.

Description

Inclusion Criteria:

  1. The subjects or their legal guardians voluntarily sign the informed consent, and can complete the study in accordance with the requirements of the program;
  2. No age or gender restriction;
  3. Patients with blood disease confirmed by histopathology or cytology;
  4. BU-based regimen will be adopted. The BU administration regimen was four times a day (Q6h), continuous intravenous infusion for 2 hours, 3 or 4 days in a row.

Exclusion Criteria:

  1. Patients with difficulty in vein blood sampling (if there is a needle, blood history);
  2. BU TDM was not performed during the treatment;
  3. Subjects who are considered unfit to participate in the trail by the investigator.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
event-free survival (EFS)
Time Frame: 2 years after administration of busulfan
EFS is calculated from the time of transplant until death, relapse of disease, or graft failure (defined as non-engraftment or rejection), whichever occurred first.
2 years after administration of busulfan
overall survival (OS)
Time Frame: 2 years after administration of busulfan
OS is the time between transplantation and death of any cause.
2 years after administration of busulfan

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
transplantation-related mortality (TRM)
Time Frame: 2 years after administration of busulfan
TRM is defined as death unrelated to underlying disease.
2 years after administration of busulfan
acute toxicity
Time Frame: 2 years after administration of busulfan
Acute toxicities are defined as VOD (diagnosed according to modified Seattle criteria),, acute GvHD grade II-IV (diagnosed and graded according to Mount Sinai Acute GvHD International Consortium (MAGIC) criteria, oral mucositis (grade II-IV) and hemorrhagic cystitis (grade II-IV). Two of the most commonly utilised scales for oral mucositis are the WHO and NCI-CTCAE scales.
2 years after administration of busulfan
chronic GvHD
Time Frame: 2 years after administration of busulfan
Chronic GvHD should be graded as mild, moderate or severe according to the National Institutes of Health (NIH) consensus criteria
2 years after administration of busulfan

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The First Affiliated Hospital of Soochow University

Collaborators

The First Affiliated Hospital of Zhengzhou University
Children's Hospital of Fudan University
Fujian Medical University Union Hospital
Beijing Children's Hospital
Children's Hospital of Soochow University
West China Second University Hospital
Guangzhou Women and Children's Medical Center
Shenzhen Children's Hospital

Investigators

  • Principal Investigator: liyan Miao, PhD, the First Affiliated Hospital of Soochow University
  • Principal Investigator: Shaoyan Hu, MD, Children's Hospital of Soochow University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 1, 2020

Primary Completion (Anticipated)

December 1, 2022

Study Completion (Anticipated)

June 1, 2023

Study Registration Dates

First Submitted

March 3, 2021

First Submitted That Met QC Criteria

March 5, 2021

First Posted (Actual)

March 8, 2021

Study Record Updates

Last Update Posted (Actual)

March 10, 2021

Last Update Submitted That Met QC Criteria

March 8, 2021

Last Verified

March 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2019-KY-BXA-01

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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