Haploidentical Stem Cell Transplantation for Children With Acquired Severe Aplastic Anemia

December 30, 2012 updated by: Ho Joon Im, Asan Medical Center

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

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

Study Overview

Study Type

Interventional

Enrollment (Anticipated)

10

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

      • Seoul, Korea, Republic of, 138-736
        • Recruiting
        • Asan Medical Center
        • Contact:
        • Sub-Investigator:
          • Kyung Nam Koh, MD, PhD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 19 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: HAPLO
Other Names:
  • Beginning on day 4 and continuing until blood counts recover
40mg/M2 once daily IV on days -7 to -4
60 mg/kg IV on day-3 and -2
200 cGy per day on D-5 & -4
Immunogenetic depletion on CliniMACS

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Time Frame: 2 years posttransplant
2 years posttransplant

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: Ho Joon Im, MD, PhD, Asan Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2012

Primary Completion (Anticipated)

December 1, 2014

Study Completion (Anticipated)

December 1, 2015

Study Registration Dates

First Submitted

December 30, 2012

First Submitted That Met QC Criteria

December 30, 2012

First Posted (Estimate)

January 3, 2013

Study Record Updates

Last Update Posted (Estimate)

January 3, 2013

Last Update Submitted That Met QC Criteria

December 30, 2012

Last Verified

December 1, 2012

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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