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

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified November 2013 by Seoul National University Hospital.
Recruitment status was:  Recruiting
Information provided by:
Seoul National University Hospital Identifier:
First received: March 4, 2011
Last updated: November 17, 2013
Last verified: November 2013
In this study the investigators plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.

Condition Intervention Phase
Chronic Granulomatous Disease
Drug: Busulfan
Phase 1
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by Seoul National University Hospital:

Primary Outcome Measures:
  • Engraftment rate [ Time Frame: 1 month after transplantation ]
    To evaluate engraftment rate after hematopoietic stem cell transplantation.

Secondary Outcome Measures:
  • Transplantation-related mortality and toxicities [ Time Frame: 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.

Estimated Enrollment: 5
Study Start Date: January 2011
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 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
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

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.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
  Contacts and Locations
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Please refer to this study by its identifier: NCT01338675

Korea, Republic of
Seoul National University Hospital
Seoul, Daehangno, Jongno-gu, Korea, Republic of
Sponsors and Collaborators
Seoul National University Hospital
Principal Investigator: Hyoung Jin Kang, M.D., Ph.D Seoul National University Hospital
  More Information Identifier: NCT01338675     History of Changes
Other Study ID Numbers: SNUCH-SCT -1002 
Study First Received: March 4, 2011
Last Updated: November 17, 2013

Keywords provided by Seoul National University Hospital:
Chronic granulomatous disease
Hematopoietic Stem Cell Transplantation

Additional relevant MeSH terms:
Granulomatous Disease, Chronic
Lymphoproliferative Disorders
Lymphatic Diseases
Pathologic Processes
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Immune System Diseases
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Antineoplastic Agents
Myeloablative Agonists processed this record on February 20, 2017