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Completed

NCT Number: NCT01105273

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

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.

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Key information

Conditions

Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Age range

Up to 21 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Asan Medical Center

Seoul, 138-736, South Korea

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

anti-thymocyte globulin

Biological

On days -3 to -1

filgrastim

Biological

Beginning on day 4 and continuing until blood counts recover

Fludarabine

Drug

30mg/M2 once daily IV on days -6 to -2

Cyclophosphamide

Drug

60 mg/kg IV on day-3 and -2

CD3±CD19 depleted hematopoietic stem cell transplantation

Procedure

Immunogenetic depletion on CliniMACS

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

Asan Medical Center

Other

Registry information

Official study title

HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3±CD19 Depletion for Patients With Aplastic Anemia After Conditioning of Fludarabine, Cyclophosphamide and Antithymocyte Globulin

Important dates

Study start
2009
Primary completion
2012
Study completion
2012
First posted
Apr 16, 2010
Registry last updated
Jan 3, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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