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

Chronic Granulomatous Disease Clinical Trial

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01338675
• Other study ID #: SNUCH-SCT -1002
• Status: Recruiting
• Phase: Phase 1/Phase 2
• First received: March 4, 2011
• Last updated: November 17, 2013
• Start date: January 2011
• Est. completion date: December 2013

Study information

• Verified date: November 2013
• Source: Seoul National University Hospital
• Contact: Hyoung Jin Kang, MD, PhD
• Phone: 82 2 2072 3304
• Email: kanghj[@]snu.ac.kr
• Is FDA regulated: No
• Health authority: Korea: Food and Drug AdministrationKorea: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 5
• Est. completion date: December 2013
• Est. primary completion date: December 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Chronic Granulomatous Disease
• Granuloma
• Granulomatous Disease, Chronic

Intervention

Drug:
• 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

Locations

Country	Name	City	State
Korea, Republic of	Seoul National University Hospital	Seoul	Daehangno, Jongno-gu

Sponsors (1)

Lead Sponsor	Collaborator
Seoul National University Hospital

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Engraftment rate	To evaluate engraftment rate after hematopoietic stem cell transplantation.	1 month after transplantation	Yes
Secondary	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.	1, 3, 6 and 12 months after transplantation	Yes