---
title: Precision Dosing of Busulfan in Children Undergoing HSCT
nct_id: NCT04822532
overall_status: UNKNOWN
phase: NA
sponsor: University Hospital, Geneva
study_type: INTERVENTIONAL
primary_condition: Hematopoietic Stem Cell Transplantation
countries: Switzerland
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT04822532.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT04822532"
ct_last_update_post_date: 2021-08-02
last_seen_at: "2026-05-12T07:25:09.385Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Precision Dosing of Busulfan in Children Undergoing HSCT

**Official Title:** Implementing Pharmacogenetics in the Busulfan Dosing Method for Children Undergoing Hematopoietic Stem-cell Transplantation: a Prospective, Multicentric, Randomized Clinical Trial

**NCT ID:** [NCT04822532](https://clinicaltrials.gov/study/NCT04822532)

## Key Facts

- **Status:** UNKNOWN
- **Phase:** NA
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 260
- **Lead Sponsor:** University Hospital, Geneva
- **Conditions:** Hematopoietic Stem Cell Transplantation, Allogeneic Hematopoietic Stem Cell Transplantation, Autologous Hematopoietic Stem Cell Transplantation
- **Start Date:** 2021-06-15
- **Completion Date:** 2025-06
- **CT.gov Last Update:** 2021-08-02

## Brief 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.

## Detailed 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).

## Eligibility

- **Maximum age:** 18 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
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)
```

## Arms

- **Pharmacogenetic based-model (GSTA1)** (EXPERIMENTAL)
- **The most performing method based on age and weight - McCune's model** (ACTIVE_COMPARATOR)

## Interventions

- **GSTA1 genotyping** (GENETIC) — Diplotype determination based on 4 single-nucleotide polymorphisms (SNPs) occurring in the promoter region of the GSTA1 gene

## Primary Outcomes

- **Accuracy of the first-dose Bu area under the curve (AUC) prediction** _(time frame: 1 month)_ — Proportion of the first doses which result in AUCs within the therapeutic target range defined by the prescriber
- **Accuracy of the Bu Clearance prediction** _(time frame: 1 month)_ — Absolute prediction error between the predicted and measured Bu clearance of the first dose
- **Dose adjustment requirement** _(time frame: 1 month)_ — Change in percentage between the first dose administered and the next time-wise adjustable dose: 2nd (Bu q24h), 3rd (Bu q12h), or 5th (Bu q6h) doses

## Secondary Outcomes

- **Time to deliver the personalized dose** _(time frame: 1 week)_
- **Incidence of treatment-related toxicities (TRTs)** _(time frame: 12 months)_
- **Incidence and severity of sinusoidal obstruction syndrome (SOS)** _(time frame: 12 months)_
- **Incidence of primary and secondary graft failure** _(time frame: 12 months)_
- **Incidence and severity of acute graft-versus-host disease (aGVHD)** _(time frame: 12 months)_
- **Overall survival** _(time frame: 12 months)_
- **Event-free survival** _(time frame: 12 months)_

## Locations (1)

- Hôpitaux Universitaires de Genève, Geneva, Switzerland — _RECRUITING_

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.maxAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.hôpitaux universitaires de genève|geneva||switzerland` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT04822532.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT04822532*  
*This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*