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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01105273
Recruitment Status : Completed
First Posted : April 16, 2010
Last Update Posted : January 3, 2013
Information provided by (Responsible Party):
Ho Joon Im, Asan Medical Center

Brief Summary:

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 or disease Intervention/treatment 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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
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
Study Start Date : July 2009
Actual Primary Completion Date : July 2012
Actual Study Completion Date : July 2012

Resource links provided by the National Library of Medicine

Drug Information available for: Fludarabine

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. 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 :
  1. To assess engraftment and graft failure [ Time Frame: 28 days post-transplant ]
    Number of patients who failed to engraft by 28 days.

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

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

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

Information from the National Library of Medicine

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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

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 identifier (NCT number): NCT01105273

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

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Ho Joon Im, Professor, Asan Medical Center Identifier: NCT01105273    
Other Study ID Numbers: AMCPHO-SCT0802
First Posted: April 16, 2010    Key Record Dates
Last Update Posted: January 3, 2013
Last Verified: December 2012
Keywords provided by Ho Joon Im, Asan Medical Center:
Aplastic anemia
CD3±CD19 depletion
Haploidentical hematopoietic stem cell transplantation
Additional relevant MeSH terms:
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Anemia, Aplastic
Hematologic Diseases
Bone Marrow Failure Disorders
Bone Marrow Diseases
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