Haploidentical Stem Cell Transplantation for Children With Acquired Severe Aplastic Anemia

Phase 2

• Conditions: Acquired Aplastic Anemia
• Interventions: Drug: Fludarabine; Drug: Cyclophosphamide; Biological: anti-thymocyte globulin; Biological: filgrastim; Radiation: Total body irradiation; Procedure: CD3-depleted hematopoietic cell transplantation

• Registration Number: NCT01759732
• Lead Sponsor: Asan Medical Center

Brief Summary

Rationale: Fludarabine, cyclophosphamide, anti-thymocyte globulin and low-dose total body irradiation (LD-TBI) may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. In addition, depletion of CD3 cells may contribute to prevent developing severe acute graft versus host disease (GVHD) in haploidentical transplantation.

Purpose: Phase II trials to evaluate the efficacy of haploidentical stem cell transplantation with fixed dose of T cells after in vitro T cell depletion using CD3 monoclonal antibody for children with acquired severe aplastic anemia

Detailed Description

Not available

Study Timeline

• Study Start: 2012-09
• Status Verified: 2012-12
• Study First Submitted: 2012-12-30
• Study First Submitted QC: 2012-12-30
• Last Update Submitted: 2012-12-30
• Study First Posted: 2013-01-03
• Last Update Posted: 2013-01-03
• Primary Completion: 2014-12
• Study Completion: 2015-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 10

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
HAPLO	anti-thymocyte globulin	-
HAPLO	filgrastim	-
HAPLO	Total body irradiation	-
HAPLO	CD3-depleted hematopoietic cell transplantation	-
HAPLO	Fludarabine	-
HAPLO	Cyclophosphamide	-

Primary Outcome Measures

Name	Time	Method
To assess engraftment rate and survival of haploidentical stem cell transplantation with fixed dose of T cells after in vitro T cell depletion using CD3 monoclonal antibody for children with acquired severe aplastic anemia	2 years posttransplant

Secondary Outcome Measures

Name	Time	Method
To assess engraftment and graft failure	28 days posttransplant	Number of patients who failed to engraft by 28 days
To estimate the risk of acute GVHD	100 days posttransplant	Number of patients with acute GVHD.
To assess treatment related mortality	100 days posttransplant	Number of death after transplantation
To estimate overall survival and failure free survival	1 year posttransplant

Trial Locations

Locations (1)

Asan Medical Center; 🇰🇷 Seoul, Korea, Republic of