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

Sponsor
Asan Medical Center (Other)
Overall Status
Unknown status
CT.gov ID
NCT01509300
Collaborator
(none)
10
Enrollment
1
Location
1
Arm
26
Duration (Months)
0.4
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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 or DiseaseIntervention/TreatmentPhase
Phase 1/Phase 2

Study Design

Study Type:
Interventional
Anticipated Enrollment :
10 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (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
Study Start Date :
Jan 1, 2012
Anticipated Primary Completion Date :
Dec 1, 2013
Anticipated Study Completion Date :
Mar 1, 2014

Arms and Interventions

ArmIntervention/Treatment
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

Outcome Measures

Primary Outcome Measures

  1. Transplantation-related mortality and overall survival of TBI, Fludarabine, Cyclophosphamide and anti-thymocyte globulin for engraftment of CD3 depleted haploidentical peripheral blood stem cells. [2 years post-transplant]

Secondary Outcome Measures

  1. Engraftment and graft failure rates [28 days engraftment and graft failure]

    Number of patients who failed to stable engraftment by 28 days

  2. Incidence of acute GVHD [100 days post-transplant]

    Number of patients with acute GVHD.

  3. Treatment related mortality [100 days post-transplant]

    Number of death after transplantation

  4. Relapse rate and overall survival [2 year after transplantation]

Eligibility Criteria

Criteria

Ages Eligible for Study:
N/A to 21 Years
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)

  1. No HLA-identical family member or closely matched (8 or 7 of 8 HLA-locus match) unrelated marrow donor available

  2. HLA-haploidentical related donor available

Exclusion criteria

  1. Active fungal infections

  2. HIV positive

  3. Pregnant or nursing

Contacts and Locations

Locations

SiteCityStateCountryPostal Code
1Asan Medical CenterSeoulKorea, Republic of138-736

Sponsors and Collaborators

  • Asan Medical Center

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

Responsible Party:
Ho Joon Im, Principal investigator, Asan Medical Center
ClinicalTrials.gov Identifier:
NCT01509300
Other Study ID Numbers:
  • AMCPHO-SCT0902
First Posted:
Jan 13, 2012
Last Update Posted:
Jan 13, 2012
Last Verified:
Jan 1, 2012

Study Results

No Results Posted as of Jan 13, 2012