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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Ho Joon Im, Asan Medical Center
ClinicalTrials.gov Identifier:
NCT01105273
First received: April 8, 2010
Last updated: December 30, 2012
Last verified: December 2012

April 8, 2010
December 30, 2012
July 2009
July 2012   (final data collection date for primary outcome measure)
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 ] [ Designated as safety issue: Yes ]
Same as current
Complete list of historical versions of study NCT01105273 on ClinicalTrials.gov Archive Site
  • To assess engraftment and graft failure [ Time Frame: 28 days post-transplant ] [ Designated as safety issue: Yes ]
    Number of patients who failed to engraft by 28 days.
  • To estimate the risk of acute GVHD [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]
    Number of patients with acute GVHD.
  • To assess treatment related mortality [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]
    Number of death after transplantation
  • To estimate overall survival [ Time Frame: 1 year after transplantation ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Human Leukocyte Antigen (HLA)-Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Aplastic Anemia
HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3±CD19 Depletion for Patients With Aplastic Anemia After Conditioning of Fludarabine, Cyclophosphamide and Antithymocyte Globulin

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.

Not Provided
Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Aplastic Anemia
  • 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
Experimental: HAPLO
Interventions:
  • Biological: anti-thymocyte globulin
  • Biological: filgrastim
  • Drug: Fludarabine
  • Drug: Cyclophosphamide
  • Procedure: CD3±CD19 depleted hematopoietic stem cell transplantation

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
12
July 2012
July 2012   (final data collection date for primary outcome measure)

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
Both
up to 21 Years
No
Contact information is only displayed when the study is recruiting subjects
Korea, Republic of
 
NCT01105273
AMCPHO-SCT0802
Not Provided
Ho Joon Im, Asan Medical Center
Asan Medical Center
Not Provided
Principal Investigator: Ho Joon Im, MD & PhD Asan Medical Center
Asan Medical Center
December 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP