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

March 23, 2012 updated by: The Korean Society of Pediatric Hematology Oncology

Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Bone Marrow (or Mobilized Peripheral Blood) Transplantation in Severe Aplastic Anemia

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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)

Study Type

Interventional

Enrollment (Anticipated)

30

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Korea, Republic of
      • Seoul, Korea, Republic of, 110-744
        • Seoul National University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 25 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Fludarabine
Drug: 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)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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.
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
From Jan. 1. 2006 to Dec. 31. 2008. For 3 years

Secondary Outcome Measures

Outcome Measure
Time Frame
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.
From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The Korean Society of Pediatric Hematology Oncology

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2006

Primary Completion (ANTICIPATED)

August 1, 2012

Study Completion (ANTICIPATED)

December 1, 2012

Study Registration Dates

First Submitted

August 18, 2008

First Submitted That Met QC Criteria

August 18, 2008

First Posted (ESTIMATE)

August 19, 2008

Study Record Updates

Last Update Posted (ESTIMATE)

March 26, 2012

Last Update Submitted That Met QC Criteria

March 23, 2012

Last Verified

March 1, 2012

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • KSPHO-SCT0401

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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