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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Total Enrollment

5 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-01-31

Study Completion Date

2013-12-31

Brief Summary

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

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Detailed Description

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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.

Conditions

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Chronic Granulomatous Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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

Group Type EXPERIMENTAL

Busulfan

Intervention Type DRUG

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

Interventions

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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

Intervention Type DRUG

Eligibility Criteria

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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.

Eligible Sex

ALL

Accepts Healthy Volunteers

No