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

• Status: Unknown status
• Trial end date: 2013-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Seoul National University Hospital
• Treatments: Busulfan

Criteria

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.