Clinical trial NCT02047578: Targeted Busulfan, Fludarabine, Etoposide Conditioning Regimen for HSCT in Childhood and Adolescent ALL.
Official title: Targeted Busulfan, Fludarabine, Etoposide Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Childhood and Adolescent Acute Lymphoblastic Leukemia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Acute Lymphoblastic Leukemia
Keywords: hematopoietic stem cell transplantation
Lead sponsor: Seoul National University Hospital
Sponsor class: OTHER
First posted: 2014-01-28
Enrollment: 38
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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 criteria: 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.

Primary outcomes:
measure: 1-year event free survival after hematopoietic stem cell transplantation.
timeFrame: at 1 year
Secondary outcomes:
measure: Engraftment rate
timeFrame: 1, 3, 6 and 12 months after transplantation
Other outcomes:
measure: Toxicities associated with hematopoietic stem cell transplantation
timeFrame: 1, 3, 6 and 12 months after transplantation
measure: Acute GVHD
timeFrame: 1, 3, 6 and 12 months after transplantation
measure: Chronic GVHD
timeFrame: 1, 3, 6 and 12 months after transplantation
measure: Treatment related mortality
timeFrame: 1, 3, 6 and 12 months after transplantation
measure: Relapse rate
timeFrame: 1, 3, 6 and 12 months after transplantation
Locations:
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
state: Daehangno, Jongno-gu
Study officials:
affiliation: Seoul National University Hospital
name: Hyoung Jin Kang, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: