Precision Dosing of Busulfan in Children Undergoing HSCT

  • STATUS
    Recruiting
  • End date
    Jun 11, 2025
  • participants needed
    260
  • sponsor
    University Hospital, Geneva
Updated on 11 August 2021

Summary

The objective of this clinical trial is to evaluate the personalization the conditioning regimen prior to the hematopoietic stem cell transplant (HSCT) in children and adolescents, to improve HSCT efficacy while reducing conditioning-related toxicities. Namely, we are going to compare the accuracy of two methods for determining the first dose of busulfan, one of the medicines used during the conditioning regimen. First doses will be determined based either only on anthropometric information such as age and weight or by adding a genetic factor that influences the individual ability of busulfan metabolization.

Description

Participants will be randomly assigned (1:1 ratio, stratified by conditioning regimen - the presence of fludarabine) to receive their first dose of busulfan according to:

  1. the most performing method based on age and weight - McCune's model (control arm)
  2. a method that also considers a pharmacogenetic factor (variants occurring in the promoter region of the GSTA1 gene) in association with the co-administered chemotherapeutic agent fludarabine in the dose personalization (experimental arm)

This is an international study being carried out in five countries (Canada, Italy, Switzerland, France, and Denmark).

Details
Condition Allogeneic Hematopoietic Stem Cell Transplantation, HSCT, Allo-Hematopoietic Stem Cell Transplant, Allogeneic Hematopoietic Cell Transplantation, Autologous Hematopoietic Stem Cell Transplantation, Autologous Stem Cell Transplantation, Stem Cell Transplantation, Hematopoietic Stem Cell Transplantation, Allogeneic Hematopoietic Stem Cell Transplant, Hematopoietic Stem Cell Transplant, Stem Cell Transplant
Treatment GSTA1 genotyping
Clinical Study IdentifierNCT04822532
SponsorUniversity Hospital, Geneva
Last Modified on11 August 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Patients must be aged from 0-18 years old on entry to the study
Clinical indication of allogeneic or autologous hematopoietic stem cell transplantation
The conditioning protocol must include IV Bu formulations, Busulfex (Otsuka Pharmaceutical), Busilvex (Pierre Fabre Pharma) or other European Medicines Agency (EMA) or Food and Drugs Administration (FDA) approved generic formulations regardless of the administration schedule (q6h, q12h, or q24h)
The expected length of time from recruitment to starting the conditioning regimen must be superior to 10 days
Informed written consent to participate in the study signed by the participant/parent

Exclusion Criteria

At least one of the drugs listed below scheduled to be administered in the Bu
administration days up to 24h after the last dose of Bu, whenever a washout is
not
possible
Metronidazol (required washout: 7 days)
Nalidixic acid (required washout: 7 days)
Phenytoin (required washout: 21 days)
Itraconazole (required washout: 14 days)
Ketoconazole (required washout: 7 days)
Voriconazole (required washout: 7 days)
Deferasirox (required washout: 7 days)
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