Clinical trial NCT01472055: Pharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell Transplantation
Official title: Pharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell Transplantation
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Acute Leukemia
Chronic Leukemia
Severe Aplastic Anemia
Keywords: Hematopoietic stem cell transplantation with fludarabine based conditioning
Lead sponsor: Seoul National University Hospital
Sponsor class: OTHER
Collaborators: name: Ministry of Food and Drug Safety, Korea
First posted: 2011-11-16
Enrollment: 46
Sex: ALL
Maximum age (years): 19
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to analysis of the pharmacokinetics of fludarabine for hematopoietic stem cell transplantation in pediatric patients.

Detailed description: Fludarabine is a commonly used chemotherapeutic agent for hematopoietic stem cell transplantation in children. However, no pharmacokinetic study has thus far been conducted in pediatric patients. Fludarabine can cause adverse reactions which include neurotoxicity, damage to lungs. Pharmacokinetic study is critical to predict the safety of fludarabine in pediatric patients.

Through an individualized pharmacokinetic study (which entails numerous samplings) of fludarabine in pediatric patients, this study aims to develop a sampling-minimized analysis method and study the population pharmacokinetics.

Through a pharmacokinetic analysis, we aim to evaluate whether pediatric patients show similar population pharmacokinetics of adults, for whom fludarabine is authorized, and evaluate the safety (toxicity) and efficacy (event free survival).

Eligibility criteria: Inclusion Criteria:

1. Conditioning regimen including fludarabine
2. Age: \< 19 years old
3. Functional class: ECOG 0-2
4. No loss of function of major organs. Criteria may be individualized.

   * Heart: shortening fraction \> 30%, ejection fraction \> 45%.
   * Liver: total bilirubin \< 2 ⅹ upper limit of normal; ALT \< 3 ⅹ upper limit of normal.
   * Kidney: creatinine \< 2 ⅹ normal or a creatinine clearance (GFR) \> 60 ml/min/1.73m2.
5. No active viral or fungal infection
6. Appropriate hematopoietic stem cell donor
7. Informed consent from patients' parents

Exclusion Criteria:

1. Pregnant or breast feeding
2. Disease progression due to clinical test
3. Psychiatric disease may interfere with clinical test
4. Whether attending physician consider the patient inappropriate for study enrollment

Primary outcomes:
measure: Analysis of the pharmacokinetics of fludarabine for hematopoietic stem cell transplantation in pediatric patients
timeFrame: 0hr (pre-dose), 30 min, 1, 3, 5, 8, 24hrs post-dose
Secondary outcomes:
measure: Comparative analysis of the pharmacokinetics of fludarabine in pediatric and adult patients
timeFrame: 3 years
measure: Evaluation of toxicity, event free survival according to the pharmacokinetics of fludarabine
timeFrame: 1, 3, 6 and 12 months after transplantation
measure: Evaluation of therapy related toxicity, significant adverse reaction
timeFrame: for 4 weeks
Other outcomes:
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: