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

Phase 1

• Conditions: Chronic Granulomatous Disease
• Interventions: Drug: Busulfan

• Registration Number: NCT01338675
• Lead Sponsor: Seoul National University Hospital

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.

Study Timeline

• Study Start: 2011-01
• Study First Submitted: 2011-03-04
• Study First Submitted QC: 2011-04-18
• Study First Posted: 2011-04-19
• Status Verified: 2013-11
• Last Update Submitted: 2013-11-17
• Last Update Posted: 2013-11-19
• Primary Completion: 2013-12
• Study Completion: 2013-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 5

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.

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Busulfan	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

Primary Outcome Measures

Name	Time	Method
Engraftment rate	1 month after transplantation	To evaluate engraftment rate after hematopoietic stem cell transplantation.

Secondary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Seoul National University Hospital; 🇰🇷 Seoul, Daehangno, Jongno-gu, Korea, Republic of