search
Back to results

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

Primary Purpose

Chronic Granulomatous Disease

Status
Unknown status
Phase
Phase 1
Locations
Korea, Republic of
Study Type
Interventional
Intervention
Busulfan
Sponsored by
Seoul National University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Chronic Granulomatous Disease focused on measuring Chronic granulomatous disease, Hematopoietic Stem Cell Transplantation

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

  • Seoul National University HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Busulfan

Arm Description

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

Outcomes

Primary Outcome Measures

Engraftment rate
To evaluate engraftment rate after hematopoietic stem cell transplantation.

Secondary Outcome Measures

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.

Full Information

First Posted
March 4, 2011
Last Updated
November 17, 2013
Sponsor
Seoul National University Hospital
search

1. Study Identification

Unique Protocol Identification Number
NCT01338675
Brief Title
Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)
Study Type
Interventional

2. Study Status

Record Verification Date
November 2013
Overall Recruitment Status
Unknown status
Study Start Date
January 2011 (undefined)
Primary Completion Date
December 2013 (Anticipated)
Study Completion Date
December 2013 (Anticipated)

3. Sponsor/Collaborators

Name of the Sponsor
Seoul National University Hospital

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chronic Granulomatous Disease
Keywords
Chronic granulomatous disease, Hematopoietic Stem Cell Transplantation

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Busulfan
Arm Type
Experimental
Arm Description
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
Intervention Name(s)
Busulfan
Intervention Description
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 Measure Information:
Title
Engraftment rate
Description
To evaluate engraftment rate after hematopoietic stem cell transplantation.
Time Frame
1 month after transplantation
Secondary Outcome Measure Information:
Title
Transplantation-related mortality and toxicities
Description
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.
Time Frame
1, 3, 6 and 12 months after transplantation

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Hyoung Jin Kang, MD, PhD
Phone
82 2 2072 3304
Email
kanghj@snu.ac.kr
First Name & Middle Initial & Last Name or Official Title & Degree
Ji Won Lee, MD
Phone
82 2 2072 0177
Email
agnesjw@hanmail.net
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hyoung Jin Kang, M.D., Ph.D
Organizational Affiliation
Seoul National University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Seoul National University Hospital
City
Seoul
State/Province
Daehangno, Jongno-gu
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hyoung Jin Kang, MD, PhD
Phone
82 2 2072 3304
Email
kanghj@snu.ac.kr
First Name & Middle Initial & Last Name & Degree
Ji Won Lee, MD
Phone
82 2 2072 0177
Email
agnesjw@hanmail.net

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs