Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)
This study is currently recruiting participants.
Verified February 2013 by Seoul National University Hospital
Sponsor:
Seoul National University Hospital
Information provided by:
Seoul National University Hospital
ClinicalTrials.gov Identifier:
NCT01338675
First received: March 4, 2011
Last updated: February 27, 2013
Last verified: February 2013
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
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:
Genetics Home Reference related topics:
chronic granulomatous disease
complement factor I deficiency
Drug Information available for:
Busulfan
U.S. FDA Resources
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
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: | February 27, 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 Phagocyte Bactericidal Dysfunction Leukocyte Disorders Hematologic Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Immunologic Deficiency Syndromes Immune System Diseases Lymphoproliferative Disorders Lymphatic Diseases Pathologic Processes |
Busulfan Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Pharmacologic Actions Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Therapeutic Uses Myeloablative Agonists |
ClinicalTrials.gov processed this record on May 21, 2013