Overview

This trial is active, not recruiting.

Condition anemia, aplastic
Treatment cyclophosphamide, fludarabine , thymoglobulin
Phase phase 2
Sponsor The Korean Society of Pediatric Hematology Oncology
Start date January 2006
End date August 2012
Trial size 30 participants
Trial identifier NCT00737685, KSPHO-SCT0401

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
cyclophosphamide, fludarabine , thymoglobulin
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 Outcomes

Measure
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.
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 Outcomes

Measure
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.

Eligibility Criteria

Male or female participants up to 25 years old.

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.

Additional Information

Official title Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Bone Marrow (or Mobilized Peripheral Blood) Transplantation in Severe Aplastic Anemia
Principal investigator Hyo Seop Ahn, M.D, Ph.D
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)
Trial information was received from ClinicalTrials.gov and was last updated in March 2012.
Information provided to ClinicalTrials.gov by The Korean Society of Pediatric Hematology Oncology.