Hematopoietic Stem Cell Transplantation With Targeted Busulfan and Fludarabine in Pediatric AML

Phase 2

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

• Registration Number: NCT01274195
• Lead Sponsor: Seoul National University Hospital

Brief Summary

In this study we plan to improve the outcome of hematopoietic stem cell transplantation by using 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 the investigators plan to improve the outcome of hematopoietic stem cell transplantation by using optimal busulfan dose through pharmacokinetic study.

Study Timeline

• Study Start: 2011-01
• Study First Submitted: 2011-01-10
• Study First Submitted QC: 2011-01-10
• Study First Posted: 2011-01-11
• Status Verified: 2013-11
• Last Update Submitted: 2013-11-17
• Last Update Posted: 2013-11-19
• Primary Completion: 2013-12
• Study Completion: 2013-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 27

Inclusion Criteria

1. Patients who are diagnosed as AML.
2. Patients who need hematopoietic stem cell transplantation with busulfan based conditioning regimen.
3. Age: up to 21 years
4. Performance status: ECOG 0-2.
5. 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.
6. Patients must lack any active viral infections or active fungal infection.
7. Appropriate donor is available: Matched in 6/6 of A, B, DR loci.
8. Patients (or one of parents if patients age < 20) 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	Busulfan	-

Primary Outcome Measures

Name	Time	Method
To evaluate 1-year event free survival after hematopoietic stem cell transplantation.	For 4 days used Buslufan through pharmacokinetic study in hematopoietic stem cell transplantation	To evaluate 1-year event free survival after hematopoietic stem cell transplantation.

Secondary Outcome Measures

Name	Time	Method
To evaluate engraftment rate, toxicities associated with hematopoietic stem cell transplantation, acute and chronic GVHD, treatment related mortality and relapse rate.	1, 3, 6 and 12 months after transplantation	To evaluate engraftment rate, toxicities associated with hematopoietic stem cell transplantation, acute and chronic GVHD, treatment related mortality and relapse rate.

Trial Locations

Locations (1)

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