Targeted Busulfan, Fludarabine, Etoposide Conditioning Regimen for HSCT in Childhood and Adolescent ALL.

Sponsor
Seoul National University Hospital (Other)
Overall Status
Recruiting
CT.gov ID
NCT02047578
Collaborator
(none)
38
Enrollment
1
Location
1
Arm
114.8
Anticipated Duration (Months)
0.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

To evaluate the outcome of hematopoietic stem cell transplantation using targeted busulfan, fludarabine, etoposide conditioning regimen in childhood and adolescent ALL.

Condition or DiseaseIntervention/TreatmentPhase
Phase 2

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 Design

Study Type:
Interventional
Anticipated Enrollment :
38 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Targeted Busulfan, Fludarabine, Etoposide Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Childhood and Adolescent Acute Lymphoblastic Leukemia
Actual Study Start Date :
Feb 5, 2014
Anticipated Primary Completion Date :
Sep 1, 2021
Anticipated Study Completion Date :
Sep 1, 2023

Arms and Interventions

ArmIntervention/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

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

Outcome Measures

Primary Outcome Measures

  1. 1-year event free survival after hematopoietic stem cell transplantation. [at 1 year]

    To evaluate 1-year event free survival after hematopoietic stem cell transplantation.

Secondary Outcome Measures

  1. Engraftment rate [1, 3, 6 and 12 months after transplantation]

    To evaluate engraftment rate

Other Outcome Measures

  1. Toxicities associated with hematopoietic stem cell transplantation [1, 3, 6 and 12 months after transplantation]

    To evaluate toxicities associated with hematopoietic stem cell transplantation. Toxicities will be assessed with number of participants.

  2. Acute GVHD [1, 3, 6 and 12 months after transplantation]

    To evaluate acute GVHD

  3. Chronic GVHD [1, 3, 6 and 12 months after transplantation]

    To evaluate chronic GVHD

  4. Treatment related mortality [1, 3, 6 and 12 months after transplantation]

    To evaluate treatment related mortality

  5. Relapse rate [1, 3, 6 and 12 months after transplantation]

    To evaluate relapse rate

Eligibility Criteria

Criteria

Ages Eligible for Study:
N/A to 21 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
  1. Patients who are diagnosed as ALL.

  2. Patients who need hematopoietic stem cell transplantation

  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 < 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.

Contacts and Locations

Locations

SiteCityStateCountryPostal Code
1Seoul National University HospitalSeoulDaehangno, Jongno-guKorea, Republic of

Sponsors and Collaborators

  • Seoul National University Hospital

Investigators

  • Principal Investigator: Hyoung Jin Kang, MD, PhD, Seoul National University Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Seoul National University Hospital
ClinicalTrials.gov Identifier:
NCT02047578
Other Study ID Numbers:
  • SNUCH_ALL_BuFluVP
First Posted:
Jan 28, 2014
Last Update Posted:
Jun 16, 2021
Last Verified:
Mar 1, 2021
Individual Participant Data (IPD) Sharing Statement:
Undecided
Plan to Share IPD:
Undecided
Keywords provided by Seoul National University Hospital
Additional relevant MeSH terms:

Study Results

No Results Posted as of Jun 16, 2021