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Human Leukocyte Antigen (HLA)-Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Aplastic Anemia

This study has been completed.
Information provided by (Responsible Party):
Ho Joon Im, Asan Medical Center Identifier:
First received: April 8, 2010
Last updated: December 30, 2012
Last verified: December 2012

Rationale: Chemotherapy with fludarabine, cyclophosphamide and anti-thymocyte globulin may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. In addition, depletion CD3±CD19 cells may contribute to prevent developing severe acute graft versus host disease (GVHD) in haploidentical transplantation.

Purpose: This phase I/II trial is to evaluate the safety and efficacy of fludarabine, cyclophosphamide and antithymocyte globulin with CD3±CD19 depleted graft from haploidentical donors in treating patients with aplastic anemia.

Condition Intervention Phase
Aplastic Anemia Biological: anti-thymocyte globulin Biological: filgrastim Drug: Fludarabine Drug: Cyclophosphamide Procedure: CD3±CD19 depleted hematopoietic stem cell transplantation Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3±CD19 Depletion for Patients With Aplastic Anemia After Conditioning of Fludarabine, Cyclophosphamide and Antithymocyte Globulin

Resource links provided by NLM:

Further study details as provided by Ho Joon Im, Asan Medical Center:

Primary Outcome Measures:
  • To assess the engraftment rate and survival of CD3±CD19 depleted haploidentical peripheral blood stem cell transplantation after conditioning with fludarabine, cyclophosphamide and anti-thymocyte globulin. [ Time Frame: 2 years post-transplant ]

Secondary Outcome Measures:
  • To assess engraftment and graft failure [ Time Frame: 28 days post-transplant ]
    Number of patients who failed to engraft by 28 days.

  • To estimate the risk of acute GVHD [ Time Frame: 100 days post-transplant ]
    Number of patients with acute GVHD.

  • To assess treatment related mortality [ Time Frame: 100 days post-transplant ]
    Number of death after transplantation

  • To estimate overall survival [ Time Frame: 1 year after transplantation ]

Enrollment: 12
Study Start Date: July 2009
Study Completion Date: July 2012
Primary Completion Date: July 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: HAPLO Biological: anti-thymocyte globulin
On days -3 to -1
Biological: filgrastim
Beginning on day 4 and continuing until blood counts recover
Drug: Fludarabine
30mg/M2 once daily IV on days -6 to -2
Drug: Cyclophosphamide
60 mg/kg IV on day-3 and -2
Procedure: CD3±CD19 depleted hematopoietic stem cell transplantation
Immunogenetic depletion on CliniMACS


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

Inclusion Criteria:

  • Diagnosis of life-threatening marrow failure (severe aplastic anemia) of nonmalignant etiology meeting 2 of the following criteria:

    • Granulocyte count < 500/mm3,
    • Corrected reticulocyte count < 1%,
    • Platelet count < 20,000/mm3
  • No HLA-identical family member or closely matched (8 of 8 HLA-locus match) unrelated marrow donor available
  • HLA-haploidentical related donor available

Exclusion Criteria:

  • Paroxysmal nocturnal hemoglobinuria or Fanconi anemia
  • Clonal cytogenetic abnormalities or myelodysplastic syndromes
  • Active fungal infections
  • HIV positive
  • Severe disease other than aplastic anemia that would severely limit the probability of survival during the graft procedure
  • Pregnant or nursing
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Please refer to this study by its identifier: NCT01105273

Korea, Republic of
Asan Medical Center
Seoul, Korea, Republic of, 138-736
Sponsors and Collaborators
Asan Medical Center
Principal Investigator: Ho Joon Im, MD & PhD Asan Medical Center
  More Information

Additional Information:
Anemia  This link exits the site


Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Ho Joon Im, Professor, Asan Medical Center Identifier: NCT01105273     History of Changes
Other Study ID Numbers: AMCPHO-SCT0802
Study First Received: April 8, 2010
Last Updated: December 30, 2012

Keywords provided by Ho Joon Im, Asan Medical Center:
Aplastic anemia
CD3±CD19 depletion
Haploidentical hematopoietic stem cell transplantation

Additional relevant MeSH terms:
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases
Fludarabine phosphate
Antilymphocyte Serum
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Antimetabolites, Antineoplastic
Antiviral Agents
Anti-Infective Agents processed this record on August 22, 2017