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

Phase 1

Completed

• Conditions: Aplastic Anemia
• Interventions: Biological: anti-thymocyte globulin; Biological: filgrastim; Drug: Fludarabine; Drug: Cyclophosphamide; Procedure: CD3±CD19 depleted hematopoietic stem cell transplantation

• Registration Number: NCT01105273
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2009-07
• Study First Submitted: 2010-04-08
• Study First Submitted QC: 2010-04-15
• Study First Posted: 2010-04-16
• Primary Completion: 2012-07
• Study Completion: 2012-07
• Status Verified: 2012-12
• Last Update Submitted: 2012-12-30
• Last Update Posted: 2013-01-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

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	Cyclophosphamide	-
HAPLO	CD3±CD19 depleted hematopoietic stem cell transplantation	-
HAPLO	Fludarabine	-

Primary Outcome Measures

Name	Time	Method
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.	2 years post-transplant

Secondary Outcome Measures

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

Trial Locations

Locations (1)

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