Hematopoietic Stem Cell Transplantation Based on Pharmacokinetic and Pharmacodynamic Modeling

Determination of Optimal Busulfan Dose Using Pharmacokinetic Modeling in Hematopoietic Stem Cell Transplantation

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01018446

Enrollment

Registered

2009-11-23

Start date

2008-12-31

Completion date

2012-02-29

Last updated

2013-11-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hematopoietic Stem Cell Transplantation

Keywords

Hematopoietic stem cell transplantation with busulfan based conditioning

Brief summary

In this study the investigators plan to develop a method to determine optimal busulfan dose through pharmacokinetic study.

Detailed description

Busulfan is a highly toxic drug with narrow therapeutic window used for the conditioning of hematopoietic stem cell transplantation. High exposure is associated with systemic toxicity such as veno-occlusive disease (VOD) and underexposure is associated with graft failure or relapse. In this study we plan to develop a method to determine optimal busulfan dose through pharmacokinetic study.

Interventions

DRUGBusulfan

First dose: busulfan (120 mg/m2 ivs once daily) (if age\<1 yr: 80 mg/ m2) Second to forth dose: according to the daily pharmacokinetic study

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 19 Years

Healthy volunteers

Inclusion criteria

1. Hematopoietic stem cell transplantation with busulfan based conditioning 2. Age: no limits. 3. Performance status: ECOG 0-2. 4. Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases. 1. Heart: a shortening fraction \> 30% and ejection fraction \> 45%. 2. Liver: total bilirubin \< 2 × upper limit of normal; ALT \< 3 × upper limit of normal. 3. Kidney: creatinine \<2 × normal or a creatinine clearance (GFR) \> 60 ml/min/1.73m2. 5. Patients must lack any active viral infections or active fungal infection. 6. Appropriate donor is available: Matched in 6/6 of A, B, DR loci. 7. Patients (or one of parents if patients age \< 19) should sign informed consent.

Exclusion criteria

1. Pregnant or nursing women. 2. Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy. 3. Psychiatric disorder that would preclude compliance.

Design outcomes

Primary

Measure	Time frame
To develop a method to determine optimal dose of busulfan through pharmacokinetic study in hematopoietic stem cell transplantation.	For 4 days used Buslufan through pharmacokinetic study in hematopoietic stem cell transplantation

Secondary

Measure	Time frame
To evaluate survival, toxicities, engraftment rate of patients received optimal dose of busulfan.	1, 3, 6 and 12 months after transplantation

Countries

South Korea

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026