Back to resultsFlu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blood)in Severe Aplastic Anemia (SAA)
Primary Purpose
Anemia, Aplastic
Status
Unknown status
Phase
Phase 2
Locations
Korea, Republic of
Study Type
Interventional
Intervention
cyclophosphamide, fludarabine , thymoglobulin
Sponsored by
About this trial
This is an interventional treatment trial for Anemia, Aplastic focused on measuring Pediatric, UBMT, Severe
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria
- and either marrow criterion.
Peripheral blood
- Neutrophils < 0.5 x 109/l
- Platelets < 20 x 109/l
- Corrected reticulocytes < 1%
Bone marrow
- Severe hypocellularity (< 25%)
- Moderate hypocellularity (25-30%) with hematopoietic cells representing < 30% of residual cells
- No prior hematopoietic stem cell transplantation.
- Age: no limits.
- 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%, 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: Matched in 6/6 of A, B, DR loci.
- Patients (or one of parents if patients age < 19) should sign informed consent.
Exclusion Criteria:
- Pregnant or nursing women.
- Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.
- Psychiatric disorder that would preclude compliance.
- Congenital aplastic anemia including Fanconi anemia.
- Manipulated bone marrow.
Sites / Locations
- Seoul National University Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Fludarabine
Arm Description
Outcomes
Primary Outcome Measures
To evaluate the engraftment potential, incidence and severity of acute graft versus host disease,toxicity of conditioning regimen for UBMT in SAA.
To evaluate overall and EFS follow-up of 1 year after UBMT/PBSCT.
Secondary Outcome Measures
To evaluate chronic GVHD and immunologic recovery after UBMT/PBSCT. and the efficacy of UBMT/PBSCT before immuno-suppressive therapy with anti-thymocyte globulin in severe aplastic anemia and long term toxicity of non-TBI based conditioning
Full Information
NCT ID
NCT00737685
First Posted
August 18, 2008
Last Updated
March 23, 2012
Sponsor
The Korean Society of Pediatric Hematology Oncology
1. Study Identification
Unique Protocol Identification Number
NCT00737685
Brief Title
Flu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blood)in Severe Aplastic Anemia (SAA)
Official Title
Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Bone Marrow (or Mobilized Peripheral Blood) Transplantation in Severe Aplastic Anemia
Study Type
Interventional
2. Study Status
Record Verification Date
March 2012
Overall Recruitment Status
Unknown status
Study Start Date
January 2006 (undefined)
Primary Completion Date
August 2012 (Anticipated)
Study Completion Date
December 2012 (Anticipated)
3. Sponsor/Collaborators
Name of the Sponsor
The Korean Society of Pediatric Hematology Oncology
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
Anti-thymocyte globulin (ATG) has been used in severe aplastic anemia as a part of the conditioning regimen. Among the many kinds of ATG preparations, thymoglobulin had been found to be more effective in preventing GVHD and rejection of organ transplants. As the fludarabine based conditioning regimens without total body irradiation have been reported to be promising for BMT/PBSCT from alternative donors in SAA, thymoglobulin was added to fludarabine and cyclophosphamide conditioning to reduce GVHD and to allow good engraftment in UBMT/UPBSCT.
Detailed Description
GVHD prophylaxis recommendation tacrolimus (0.03 mg/kg/day i.v. by continuous infusion from day -2 and taper with an oral form until 1 year after BMT/PBSCT) methotrexate (15 mg/m2 i.v. on days 1 and 10 mg/m2 i.v. on days 3, 6, 11)
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Anemia, Aplastic
Keywords
Pediatric, UBMT, Severe
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
30 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Fludarabine
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
cyclophosphamide, fludarabine , thymoglobulin
Intervention Description
cyclophosphamide (50 mg/kg once daily i.v. on days -9, -8, -7 & -6) fludarabine (30 mg/m2 once daily i.v. on days -5, -4, -3 & -2) thymoglobulin (2.5 mg/kg once daily i.v. on days -3, -2 & -1)
Primary Outcome Measure Information:
Title
To evaluate the engraftment potential, incidence and severity of acute graft versus host disease,toxicity of conditioning regimen for UBMT in SAA.
Time Frame
From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.
Title
To evaluate overall and EFS follow-up of 1 year after UBMT/PBSCT.
Time Frame
From Jan. 1. 2006 to Dec. 31. 2008. For 3 years
Secondary Outcome Measure Information:
Title
To evaluate chronic GVHD and immunologic recovery after UBMT/PBSCT. and the efficacy of UBMT/PBSCT before immuno-suppressive therapy with anti-thymocyte globulin in severe aplastic anemia and long term toxicity of non-TBI based conditioning
Time Frame
From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.
10. Eligibility
Sex
All
Maximum Age & Unit of Time
25 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria
and either marrow criterion.
Peripheral blood
Neutrophils < 0.5 x 109/l
Platelets < 20 x 109/l
Corrected reticulocytes < 1%
Bone marrow
Severe hypocellularity (< 25%)
Moderate hypocellularity (25-30%) with hematopoietic cells representing < 30% of residual cells
No prior hematopoietic stem cell transplantation.
Age: no limits.
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%, 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: Matched in 6/6 of A, B, DR loci.
Patients (or one of parents if patients age < 19) should sign informed consent.
Exclusion Criteria:
Pregnant or nursing women.
Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.
Psychiatric disorder that would preclude compliance.
Congenital aplastic anemia including Fanconi anemia.
Manipulated bone marrow.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hyo Seop Ahn, M.D, Ph.D
Organizational Affiliation
The Korean Society of Pediatric Hematology Oncology
Official's Role
Principal Investigator
Facility Information:
Facility Name
Seoul National University Hospital
City
Seoul
ZIP/Postal Code
110-744
Country
Korea, Republic of
12. IPD Sharing Statement
Citations:
PubMed Identifier
20685256
Citation
Kang HJ, Shin HY, Park JE, Chung NG, Cho B, Kim HK, Kim SY, Lee YH, Lim YT, Yoo KH, Sung KW, Koo HH, Im HJ, Seo JJ, Park SK, Ahn HS; Korean Society of Pediatric Hematology-Oncology. Successful engraftment with fludarabine, cyclophosphamide, and thymoglobulin conditioning regimen in unrelated transplantation for severe aplastic anemia: A phase II prospective multicenter study. Biol Blood Marrow Transplant. 2010 Nov;16(11):1582-8. doi: 10.1016/j.bbmt.2010.05.010. Epub 2010 May 26.
Results Reference
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Flu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blood)in Severe Aplastic Anemia (SAA)
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