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

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified March 2012 by The Korean Society of Pediatric Hematology Oncology.
Recruitment status was:  Recruiting
Information provided by:
The Korean Society of Pediatric Hematology Oncology Identifier:
First received: April 14, 2009
Last updated: March 23, 2012
Last verified: March 2012
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 Intervention Phase
Aplastic Anemia
Other: Cyclophosphosphamide, TBI
Phase 1
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Parallel Assignment
Masking: 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

Resource links provided by NLM:

Further study details as provided by The Korean Society of Pediatric Hematology Oncology:

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

Estimated Enrollment: 5
Study Start Date: November 2008
Estimated Study Completion Date: September 2012
Estimated Primary Completion Date: September 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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)


Ages Eligible for Study:   1 Year to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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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Please refer to this study by its identifier: NCT00881933

Contact: Hyoung Jin Kang, M.D, Ph.D 82 2 2072 3304
Contact: Ji Won Lee, M.D 82 2 2072 0177

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   
Sponsors and Collaborators
The Korean Society of Pediatric Hematology Oncology
Principal Investigator: Hyo Seop Ahn, M.D, Ph.D The Korean Society of Pediatric Hematology Oncology
  More Information

Responsible Party: The Korean Society of Hematology, The Korean Society of Pediatric Hematology Oncology Identifier: NCT00881933     History of Changes
Other Study ID Numbers: KSPHO-SCT0804
Study First Received: April 14, 2009
Last Updated: March 23, 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:
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases
Fludarabine phosphate
Antineoplastic Agents
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antiviral Agents
Anti-Infective Agents
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Myeloablative Agonists processed this record on May 25, 2017