Hematopoietic Stem Cell Transplantation Based on Pharmacokinetic and Pharmacodynamic Modeling

Phase 1

Completed

• Conditions: Hematopoietic Stem Cell Transplantation
• Interventions: Drug: Busulfan

• Registration Number: NCT01018446
• Lead Sponsor: Seoul National University Hospital

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.

Study Timeline

• Study Start: 2008-12
• Study First Submitted: 2009-11-20
• Study First Submitted QC: 2009-11-20
• Study First Posted: 2009-11-23
• Primary Completion: 2012-02
• Study Completion: 2012-02
• Status Verified: 2013-11
• Last Update Submitted: 2013-11-17
• Last Update Posted: 2013-11-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 30

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Busulfan, Pharmacokinetic	Busulfan	To develop a method to determine optimal dose of busulfan through pharmacokinetic study in hematopoietic stem cell transplantation.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

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

Trial Locations

Locations (1)

Seoul National University Hospital; 🇰🇷 Seoul, Daehangno, Jongno-gu, Korea, Republic of