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

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

Study Overview

• Status: Unknown
• Conditions: Hematopoietic Stem Cell Transplantation
• Intervention / Treatment: Drug: Busulfan

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 Type: Interventional
• Enrollment (Anticipated): 27
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Ji Won Lee, MD; Phone Number: 82 2 2072 0177; Email: agnesjw@hanmail.net
• Study Contact Backup: Name: Hyoung Jin Kang, MD, PhD; Phone Number: 82 2 2072 0177; Email: kanghj@snu.ac.kr

Study Locations

• Korea, Republic of
  • Daehangno, Jongno-gu
    • Seoul, Daehangno, Jongno-gu, Korea, Republic of
      • Recruiting
      • Seoul National University Hospital
      • Contact: Ji Won Lee, MD; Phone Number: 82 2 2072 0177; Email: agnesjw@hanmail.net
      • Contact: Hyoung Jin Kang, MD, PhD; Phone Number: 82 2 2072 0177; Email: kanghj@snu.ac.kr
      • Principal Investigator: Hyo Seop Ahn, MD, PhD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 21 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Busulfan	Drug: Busulfan; 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate 1-year event free survival after hematopoietic stem cell transplantation.	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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate engraftment rate, toxicities associated with hematopoietic stem cell transplantation, acute and chronic GVHD, treatment related mortality and relapse rate.	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

Collaborators and Investigators

• Sponsor: Seoul National University Hospital

Study record dates

Study Major Dates

• Study Start: January 1, 2011
• Primary Completion (Anticipated): December 1, 2013
• Study Completion (Anticipated): December 1, 2013

Study Registration Dates

• First Submitted: January 10, 2011
• First Submitted That Met QC Criteria: January 10, 2011
• First Posted (Estimate): January 11, 2011

Study Record Updates

• Last Update Posted (Estimate): November 19, 2013
• Last Update Submitted That Met QC Criteria: November 17, 2013
• Last Verified: November 1, 2013

More Information

Terms related to this study

• Keywords: Hematopoietic stem cell transplantation
• Additional Relevant MeSH Terms: Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Antineoplastic Agents, Alkylating; Alkylating Agents; Myeloablative Agonists; Busulfan
• Other Study ID Numbers: SNUCH-SCT -1001