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

This study is currently recruiting participants.
Verified January 2012 by Asan Medical Center
Sponsor:
Information provided by (Responsible Party):
Ho Joon Im, Asan Medical Center
ClinicalTrials.gov Identifier:
NCT01509300
First received: January 5, 2012
Last updated: January 12, 2012
Last verified: January 2012
History of Changes
  Purpose

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: Endpoint Classification: Safety/Efficacy Study
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:

U.S. FDA Resources

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 ] [ Designated as safety issue: Yes ]

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

  • Incidence of acute GVHD [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]
    Number of patients with acute GVHD.

  • Treatment related mortality [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]
    Number of death after transplantation

  • Relapse rate and overall survival [ Time Frame: 2 year after transplantation ] [ Designated as safety issue: No ]

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

  Eligibility

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

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01509300

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    
Principal Investigator: Ho Joon Im, MD & PhD            
Sponsors and Collaborators
Asan Medical Center
Investigators
Principal Investigator: Ho Joon Im, MD & PhD Asan Medical Center
  More Information

Publications:

Responsible Party: Ho Joon Im, Principal investigator, Asan Medical Center
ClinicalTrials.gov Identifier: NCT01509300     History of Changes
Other Study ID Numbers: AMCPHO-SCT0902
Study First Received: January 5, 2012
Last Updated: January 12, 2012
Health Authority: Korea: Institutional Review Board

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

Additional relevant MeSH terms:
Leukemia
Myelodysplastic Syndromes
Preleukemia
Acute Disease
Neoplasms
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Disease Attributes
Pathologic Processes
Antilymphocyte Serum
Cyclophosphamide
Mycophenolate mofetil
Fludarabine monophosphate
 Tacrolimus
Lenograstim
Rituximab
Fludarabine
Mycophenolic Acid
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

ClinicalTrials.gov processed this record on May 16, 2013