Target Busulfan Exposure in Children With HSCT in China

Sponsor
The First Affiliated Hospital of Soochow University (Other)
Overall Status
Recruiting
CT.gov ID
NCT04786002
Collaborator
Children's Hospital Of Soochow University (Other), Children's Hospital of Fudan University (Other), Fujian Medical University Union Hospital (Other), Guangzhou Women and Children's Medical Center (Other), West China Second University Hospital (Other), Beijing Children's Hospital (Other), Shenzhen Children's Hospital (Other), The First Affiliated Hospital of Zhengzhou University (Other)
500
1
30.9
16.2

Study Details

Study Description

Brief Summary

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.

Condition or Disease Intervention/Treatment Phase

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 Design

Study Type:
Observational
Anticipated Enrollment :
500 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
The Therapeutic Window of Busulfan in Children With Haemopoietic Stem Cell Transplantation:A Multicenter Study in China
Actual Study Start Date :
Nov 1, 2020
Anticipated Primary Completion Date :
Dec 1, 2022
Anticipated Study Completion Date :
Jun 1, 2023

Outcome Measures

Primary Outcome Measures

  1. event-free survival (EFS) [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. overall survival (OS) [2 years after administration of busulfan]

    OS is the time between transplantation and death of any cause.

Secondary Outcome Measures

  1. transplantation-related mortality (TRM) [2 years after administration of busulfan]

    TRM is defined as death unrelated to underlying disease.

  2. acute toxicity [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.

  3. chronic GvHD [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

Eligibility Criteria

Criteria

Ages Eligible for Study:
0 Years to 18 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 the First Affiliated Hospital of Soochow University Suzhou Jiangsu China 215006

Sponsors and Collaborators

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

Investigators

  • Principal Investigator: Liyan Miao, PhD, The First Affiliated Hospital of Soochow University
  • Principal Investigator: Shaoyan Hu, MD, Children's Hospital Of Soochow University

Study Documents (Full-Text)

None provided.

More Information

Publications

Responsible Party:
The First Affiliated Hospital of Soochow University
ClinicalTrials.gov Identifier:
NCT04786002
Other Study ID Numbers:
  • 2019-KY-BXA-01
First Posted:
Mar 8, 2021
Last Update Posted:
Mar 10, 2021
Last Verified:
Mar 1, 2021
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by The First Affiliated Hospital of Soochow University
Additional relevant MeSH terms:

Study Results

No Results Posted as of Mar 10, 2021