Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)

Seoul National University Hospital5 enrolled

Overview

In this study the investigators plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.

Chronic granulomatous disease is one of the rare congenital immunodeficiency which can be cured by hematopoietic stem cell transplantation. Previous myeloablative conditioning regimen has problems related to the severe toxicities, and non-myeloablative conditioning regimen has the risk of graft failure. Recently, reduced-intensity myeloablative conditioning regimen with busulfan and fludarabine was used usually in leukemia patients. Busulfan is a highly toxic drug with narrow therapeutic window. In this study we plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Chronic Granulomatous Disease

Interventions

BusulfanDRUG

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

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion Criteria: * Patients who are diagnosed as CGD. * Patients who need hematopoietic stem cell transplantation with busulfan based conditioning regimen. * Age: No limit. * Performance status: ECOG 0-2. * Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases. * Heart: a shortening fraction \> 30% and ejection fraction \> 45%. * Liver: total bilirubin \< 2 × upper limit of normal; ALT \< 3 × upper limit of normal. * Kidney: creatinine \<2 × normal or a creatinine clearance (GFR) \> 60 ml/min/1.73m2. * Patients must lack any active viral infections or active fungal infection. * Appropriate donor is available. * Patients (or one of parents if patients age \< 20) should sign informed consent.

Locations (1)

Seoul National University Hospital

Seoul, Daehangno, Jongno-gu, South Korea

RECRUITING

Outcomes

Primary Outcomes

Engraftment rate

To evaluate engraftment rate after hematopoietic stem cell transplantation.

Time frame: 1 month after transplantation

Secondary Outcomes

Transplantation-related mortality and toxicities

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

Central Contacts

Hyoung Jin Kang, MD, PhD

CONTACT

82 2 2072 3304 kanghj@snu.ac.kr

Ji Won Lee, MD

CONTACT

82 2 2072 0177 agnesjw@hanmail.net

Data from ClinicalTrials.gov

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