search
Back to results

Flu+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
The Korean Society of Pediatric Hematology Oncology
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Anemia, Aplastic focused on measuring Pediatric, UBMT, Severe

Eligibility Criteria

undefined - 25 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria
  • and either marrow criterion.
  • Peripheral blood

    1. Neutrophils < 0.5 x 109/l
    2. Platelets < 20 x 109/l
    3. Corrected reticulocytes < 1%
  • Bone marrow

    1. Severe hypocellularity (< 25%)
    2. 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.

    1. Heart: a shortening fraction > 30%, ejection fraction > 45%.
    2. Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal.
    3. 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

First Posted
August 18, 2008
Last Updated
March 23, 2012
Sponsor
The Korean Society of Pediatric Hematology Oncology
search

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
derived

Learn more about this trial

Flu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blood)in Severe Aplastic Anemia (SAA)

We'll reach out to this number within 24 hrs