Haploidentical Stem Cell Transplantation for Children With Acquired Severe Aplastic Anemia

This study is currently recruiting participants. (see Contacts and Locations)
Verified December 2012 by Asan Medical Center
Sponsor:
Information provided by (Responsible Party):
Ho Joon Im, Asan Medical Center
ClinicalTrials.gov Identifier:
NCT01759732
First received: December 30, 2012
Last updated: NA
Last verified: December 2012
History: No changes posted
  Purpose

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


Condition Intervention Phase
Acquired Aplastic Anemia
Drug: Fludarabine
Drug: Cyclophosphamide
Biological: anti-thymocyte globulin
Biological: filgrastim
Radiation: Total body irradiation
Procedure: CD3-depleted hematopoietic cell transplantation
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Asan Medical Center:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • To assess engraftment and graft failure [ Time Frame: 28 days posttransplant ] [ 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 posttransplant ] [ Designated as safety issue: Yes ]
    Number of patients with acute GVHD.

  • To assess treatment related mortality [ Time Frame: 100 days posttransplant ] [ Designated as safety issue: Yes ]
    Number of death after transplantation

  • To estimate overall survival and failure free survival [ Time Frame: 1 year posttransplant ] [ Designated as safety issue: No ]

Estimated Enrollment: 10
Study Start Date: September 2012
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: HAPLO Drug: Fludarabine
40mg/M2 once daily IV on days -7 to -4
Drug: Cyclophosphamide
60 mg/kg IV on day-3 and -2
Biological: anti-thymocyte globulin Biological: filgrastim
Other Name: Beginning on day 4 and continuing until blood counts recover
Radiation: Total body irradiation
200 cGy per day on D-5 & -4
Procedure: CD3-depleted hematopoietic cell transplantation
Immunogenetic depletion on CliniMACS

  Eligibility

Ages Eligible for Study:   up to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01759732

Contacts
Contact: Ho Joon Im, MD, PhD 82-2-3010-3371 hojim@amc.seoul.kr

Locations
Korea, Republic of
Asan Medical Center Recruiting
Seoul, Korea, Republic of, 138-736
Contact: Ho Joon Im, MD, PhD    82-2-3010-3371    hojim@amc.seoul.kr   
Sub-Investigator: Kyung Nam Koh, MD, PhD         
Sponsors and Collaborators
Asan Medical Center
Investigators
Principal Investigator: Ho Joon Im, MD, PhD Asan Medical Center
  More Information

Additional Information:
Anemia  This link exits the ClinicalTrials.gov site

Publications:

Responsible Party: Ho Joon Im, Professor, Asan Medical Center
ClinicalTrials.gov Identifier: NCT01759732     History of Changes
Other Study ID Numbers: AMCPHO-SCT1202
Study First Received: December 30, 2012
Last Updated: December 30, 2012
Health Authority: Korea: Institutional Review Board

Keywords provided by Asan Medical Center:
Aplastic anemia
T cell dose
CD3 depletion
Haploidentical hematopoietic stem cell transplantation

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases
Cyclophosphamide
Antilymphocyte Serum
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists

ClinicalTrials.gov processed this record on September 22, 2014