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

Status

UNKNOWN

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01338675

Enrollment

Registered

2011-04-19

Start date

2011-01-31

Completion date

2013-12-31

Last updated

2013-11-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Chronic Granulomatous Disease

Keywords

Chronic granulomatous disease, Hematopoietic Stem Cell Transplantation

Brief summary

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

Detailed description

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.

Interventions

DRUGBusulfan

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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
Engraftment rate	1 month after transplantation	To evaluate engraftment rate after hematopoietic stem cell transplantation.

Secondary

Measure	Time frame	Description
Transplantation-related mortality and toxicities	1, 3, 6 and 12 months after transplantation	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.

Countries

South Korea

Contacts

Primary ContactHyoung Jin Kang, MD, PhD

kanghj@snu.ac.kr82 2 2072 3304

Backup ContactJi Won Lee, MD

agnesjw@hanmail.net82 2 2072 0177

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026