Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2013 by Seoul National University Hospital
Sponsor:
Information provided by:
Seoul National University Hospital
ClinicalTrials.gov Identifier:
NCT01338675
First received: March 4, 2011
Last updated: November 17, 2013
Last verified: November 2013
  Purpose

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: Endpoint Classification: Safety/Efficacy Study
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 ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: Yes ]
    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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01338675

Contacts
Contact: Hyoung Jin Kang, MD, PhD 82 2 2072 3304 kanghj@snu.ac.kr
Contact: Ji Won Lee, MD 82 2 2072 0177 agnesjw@hanmail.net

Locations
Korea, Republic of
Seoul National University Hospital Recruiting
Seoul, Daehangno, Jongno-gu, Korea, Republic of
Contact: Hyoung Jin Kang, MD, PhD    82 2 2072 3304    kanghj@snu.ac.kr   
Contact: Ji Won Lee, MD    82 2 2072 0177    agnesjw@hanmail.net   
Sponsors and Collaborators
Seoul National University Hospital
Investigators
Principal Investigator: Hyoung Jin Kang, M.D., Ph.D Seoul National University Hospital
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT01338675     History of Changes
Other Study ID Numbers: SNUCH-SCT -1002
Study First Received: March 4, 2011
Last Updated: November 17, 2013
Health Authority: Korea: Food and Drug Administration
Korea: Institutional Review Board

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

Additional relevant MeSH terms:
Granulomatous Disease, Chronic
Granuloma
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Immune System Diseases
Immunologic Deficiency Syndromes
Leukocyte Disorders
Lymphatic Diseases
Lymphoproliferative Disorders
Pathologic Processes
Phagocyte Bactericidal Dysfunction
Busulfan
Alkylating Agents
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on November 24, 2014