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

This study is currently recruiting participants.
Verified April 2013 by Seoul National University Hospital
Sponsor:
Information provided by:
Seoul National University Hospital
ClinicalTrials.gov Identifier:
NCT01274195
First received: January 10, 2011
Last updated: April 2, 2013
Last verified: April 2013
History of Changes
  Purpose

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


Condition Intervention Phase
Hematopoietic Stem Cell Transplantation
 Drug: Busulfan
 Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Hematopoietic Stem Cell Transplantation With Targeted Busulfan and Fludarabine in Pediatric AML

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Seoul National University Hospital:

Primary Outcome Measures:
  • To evaluate 1-year event free survival after hematopoietic stem cell transplantation. [ Time Frame: For 4 days used Buslufan through pharmacokinetic study in hematopoietic stem cell transplantation ] [ Designated as safety issue: Yes ]
    To evaluate 1-year event free survival after hematopoietic stem cell transplantation.


Secondary Outcome Measures:
  • To evaluate engraftment rate, toxicities associated with hematopoietic stem cell transplantation, acute and chronic GVHD, treatment related mortality and relapse rate. [ Time Frame: 1, 3, 6 and 12 months after transplantation ] [ Designated as safety issue: No ]
    To evaluate engraftment rate, toxicities associated with hematopoietic stem cell transplantation, acute and chronic GVHD, treatment related mortality and relapse rate.


Estimated Enrollment: 27
Study Start Date: January 2011
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   up to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01274195

Contacts
Contact: Hyoung Jin Kang, MD, PhD 82 2 2072 0177 kanghj@snu.ac.kr
Contact: Ji Won Lee, MD 82 2 2072 0177 agnesjw@hanmail.net

Locations
Korea, Republic of
Seoul National University Hospital Recruiting
Seoul, Daehangno, Jongno-gu, Korea, Republic of
Contact: Hyoung Jin Kang, MD, PhD     82 2 2072 0177     kanghj@snu.ac.kr    
Contact: Ji Won Lee, MD     82 2 2072 0177     agnesjw@hanmail.net    
Principal Investigator: Hyo Seop Ahn, MD, PhD            
Sponsors and Collaborators
Seoul National University Hospital
Investigators
Principal Investigator: Hyoung Jin Kang, M.D., Ph.D Seoul National University Hospital
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT01274195     History of Changes
Other Study ID Numbers: SNUCH-SCT -1001
Study First Received: January 10, 2011
Last Updated: April 2, 2013
Health Authority: Korea: Food and Drug Administration

Keywords provided by Seoul National University Hospital:
Hematopoietic stem cell transplantation

Additional relevant MeSH terms:
Busulfan
Fludarabine
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
 Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Therapeutic Uses
Myeloablative Agonists

ClinicalTrials.gov processed this record on May 16, 2013