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

Status

Completed

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01105273

Enrollment

Registered

2010-04-16

Start date

2009-07-31

Completion date

2012-07-31

Last updated

2013-01-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Aplastic Anemia

Keywords

Aplastic anemia, Fludarabine, CD3±CD19 depletion, Haploidentical hematopoietic stem cell transplantation

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.

Interventions

BIOLOGICALanti-thymocyte globulin

On days -3 to -1

BIOLOGICALfilgrastim

Beginning on day 4 and continuing until blood counts recover

DRUGFludarabine

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

DRUGCyclophosphamide

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

PROCEDURECD3±CD19 depleted hematopoietic stem cell transplantation

Immunogenetic depletion on CliniMACS

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 21 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame	Description
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	—

Countries

South Korea

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026