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HLA-haploidentical Hematopoietic Stem Cell Transplantation for Children and Adolescents With Acute Leukemia, Myelodysplastic Syndrome and Solid Tumors

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified January 2012 by Asan Medical Center.
Recruitment status was:  Recruiting
Information provided by (Responsible Party):
Ho Joon Im, Asan Medical Center Identifier:
First received: January 5, 2012
Last updated: January 12, 2012
Last verified: January 2012

RATIONALE: Conditioning with total body irradiation (TBI) and 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, T-cell depletion 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 TBI, fludarabine, cyclophosphamide and antithymocyte globulin with T-cell depleted graft from haploidentical donors in treating patients with acute leukemia and myelodysplastic syndrome.

Condition Intervention Phase
Acute Leukemia
Myelodysplastic Syndrome
Solid Tumors
Biological: anti-thymocyte globulin
Biological: filgrastim
Radiation: Total body irradiation
Drug: Fludarabine
Drug: cyclophosphamide
Drug: Tacrolimus
Drug: Mycophenolate mofetil
Drug: Rituximab
Phase 1
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3 Depletion for Children and Adolescents With Acute Leukemia, Myelodysplastic Syndrome and Solid Tumors After Conditioning of TBI, Fludarabine, Cyclophosphamide and Antithymocyte Globulin

Resource links provided by NLM:

Further study details as provided by Asan Medical Center:

Primary Outcome Measures:
  • Transplantation-related mortality and overall survival of TBI, Fludarabine, Cyclophosphamide and anti-thymocyte globulin for engraftment of CD3 depleted haploidentical peripheral blood stem cells. [ Time Frame: 2 years post-transplant ]

Secondary Outcome Measures:
  • Engraftment and graft failure rates [ Time Frame: 28 days engraftment and graft failure ]
    Number of patients who failed to stable engraftment by 28 days

  • Incidence of acute GVHD [ Time Frame: 100 days post-transplant ]
    Number of patients with acute GVHD.

  • Treatment related mortality [ Time Frame: 100 days post-transplant ]
    Number of death after transplantation

  • Relapse rate and overall survival [ Time Frame: 2 year after transplantation ]

Estimated Enrollment: 10
Study Start Date: January 2012
Estimated Study Completion Date: March 2014
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: HAPLO Biological: anti-thymocyte globulin
On days -10 to -9
Biological: filgrastim
Beginning on day 4 and continuing until blood counts recover
Radiation: Total body irradiation
2Gy D-6 to D-4
Drug: Fludarabine
30mg/M2 once daily IV on days -8 to -4
Drug: cyclophosphamide
60 mg/kg IV on day-3 and -2
Drug: Tacrolimus
begin on 0
Drug: Mycophenolate mofetil
begin on 0
Drug: Rituximab
375mg/m2 on day +21


Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria

  1. Disease characteristics

    • Acute lymphoblastic leukemia (first remission, high risk; beyond first remission; refractory)
    • Acute myeloblastic leukemia (first remission, high risk; beyond first remission; refractory)
    • Myelodysplastic syndrome
    • Solid tumors (Refractory/relapse)
  2. No HLA-identical family member or closely matched (8 or 7 of 8 HLA-locus match) unrelated marrow donor available
  3. HLA-haploidentical related donor available

Exclusion criteria

  1. Active fungal infections
  2. HIV positive
  3. 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 identifier: NCT01509300

Contact: Ho Joon Im, MD & PhD 82-2-3010-3371

Korea, Republic of
Asan Medical Center Recruiting
Seoul, Korea, Republic of, 138-736
Contact: Ho Joon Im, MD & PhD    82-2-3010-3371   
Principal Investigator: Ho Joon Im, MD & PhD         
Sponsors and Collaborators
Asan Medical Center
Principal Investigator: Ho Joon Im, MD & PhD Asan Medical Center
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Ho Joon Im, Principal investigator, Asan Medical Center Identifier: NCT01509300     History of Changes
Other Study ID Numbers: AMCPHO-SCT0902
Study First Received: January 5, 2012
Last Updated: January 12, 2012

Keywords provided by Asan Medical Center:
CD3 depletion
Children and adolescents
Haploidentical hematopoietic stem cell transplantation

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Acute Disease
Pathologic Processes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Disease Attributes
Fludarabine phosphate
Mycophenolate mofetil
Antilymphocyte Serum
Mycophenolic Acid
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents processed this record on May 23, 2017