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Study of Fludarabine + Cyclophosphamide + TBI Conditioning Regimen for Double Units Cord Blood Transplantation(CBT)in Severe Aplastic Anemia(SAA)

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ClinicalTrials.gov Identifier: NCT00881933
Recruitment Status : Unknown
Verified March 2012 by The Korean Society of Pediatric Hematology Oncology.
Recruitment status was:  Recruiting
First Posted : April 15, 2009
Last Update Posted : March 26, 2012
Sponsor:
Information provided by:
The Korean Society of Pediatric Hematology Oncology

Study Description
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Brief Summary:
Severe aplastic anemia is a fatal disease and patients without HLA matched siblings need alternative treatment option. Cord blood transplantation (CBT) has become an alternative treatment means in various diseases, but it has not been proved to be good for severe aplastic anemia. Double units CBT is proposed to have better engraftment potential and and we reported successful double units UCBT after engraftment failure with single unit with promising result. To increase the engraftment potential, fludarabine, cyclophosphamide plus TBI conditioning regimen for double units cord blood transplantation was proposed for the patient with severe aplastic anemia without HLA-matched donor.

Condition or disease Intervention/treatment Phase
Aplastic Anemia Other: Cyclophosphosphamide, TBI Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I/II Study of Fludarabine, Cyclophosphamide Plus TBI Conditioning Regimen for Double Units Cord Blood Transplantation in Severe Aplastic Anemia
Study Start Date : November 2008
Estimated Primary Completion Date : September 2012
Estimated Study Completion Date : September 2012

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Fludarabine Other: Cyclophosphosphamide, TBI
fludarabine (30 mg/m2 once daily i.v. on days -7, -6, -5, -4, -3, & -2) (if body weight < 10 kg or if age < 1 year: 1.33 mg/kg) cyclophosphamide (60 mg/kg once daily i.v. on days -9, & -8) total body irradiation (3G on days -1)


Outcome Measures
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Primary Outcome Measures :
  1. To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for cord blood transplantation (CBT) in severe aplastic anemia. [ Time Frame: From Oct 2008. to Sep 2011 ]

Secondary Outcome Measures :
  1. To evaluate the incidence and severity of toxicity and treatment related mortality [ Time Frame: From Oct 2008. to Sep 2011 ]
  2. To evaluate overall and event free survival rate [ Time Frame: From Oct 2008. to Sep 2011 ]
  3. To evaluate acute and chronic GVHD [ Time Frame: From Oct 2008. to Sep 2011 ]
  4. To evaluate immunologic recovery after CBT [ Time Frame: From Oct 2008. to Sep 2011 ]

Eligibility Criteria
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Ages Eligible for Study:   1 Year to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
  • No appropriate donor
  • Appropriate cord blood is available: matched at least in 4/6 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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00881933


Contacts
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Contact: Hyoung Jin Kang, M.D, Ph.D 82 2 2072 3304 kanghj@snu.ac.kr
Contact: Ji Won Lee, M.D 82 2 2072 0177 agnesjw@hanmail.net

Locations
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Korea, Republic of
Seoul National University Hospital Recruiting
Seoul, Korea, Republic of, 110-744
Contact: Hyo Seop Ahn, M.D, Ph.D    82 2 2072 3625    hsahn@snu.ac.kr   
Sponsors and Collaborators
The Korean Society of Pediatric Hematology Oncology
Investigators
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Principal Investigator: Hyo Seop Ahn, M.D, Ph.D The Korean Society of Pediatric Hematology Oncology
More Information
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Responsible Party: The Korean Society of Hematology, The Korean Society of Pediatric Hematology Oncology
ClinicalTrials.gov Identifier: NCT00881933    
Other Study ID Numbers: KSPHO-SCT0804
First Posted: April 15, 2009    Key Record Dates
Last Update Posted: March 26, 2012
Last Verified: March 2012
Keywords provided by The Korean Society of Pediatric Hematology Oncology:
Fludarabine, Cyclophosphamide, TBI, severe aplastic anemia,
cord blood transplantation, pediatric
Additional relevant MeSH terms:
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Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Failure Disorders
Bone Marrow Diseases