Back to resultsHLA-haploidentical Hematopoietic Stem Cell Transplantation for Children and Adolescents With Acute Leukemia, Myelodysplastic Syndrome and Solid Tumors
Primary Purpose
Acute Leukemia, Myelodysplastic Syndrome, Solid Tumors
Status
Unknown status
Phase
Phase 1
Locations
Korea, Republic of
Study Type
Interventional
Intervention
anti-thymocyte globulin
filgrastim
Total body irradiation
Fludarabine
cyclophosphamide
Tacrolimus
Mycophenolate mofetil
Rituximab
Sponsored by
About this trial
This is an interventional treatment trial for Acute Leukemia focused on measuring TBI, Fludarabine, CD3 depletion, Children and adolescents, Haploidentical hematopoietic stem cell transplantation
Eligibility Criteria
Inclusion Criteria
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)
- No HLA-identical family member or closely matched (8 or 7 of 8 HLA-locus match) unrelated marrow donor available
- HLA-haploidentical related donor available
Exclusion criteria
- Active fungal infections
- HIV positive
- Pregnant or nursing
Sites / Locations
- Asan Medical CenterRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
HAPLO
Arm Description
Outcomes
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.
Secondary Outcome Measures
Engraftment and graft failure rates
Number of patients who failed to stable engraftment by 28 days
Incidence of acute GVHD
Number of patients with acute GVHD.
Treatment related mortality
Number of death after transplantation
Relapse rate and overall survival
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01509300
Brief Title
HLA-haploidentical Hematopoietic Stem Cell Transplantation for Children and Adolescents With Acute Leukemia, Myelodysplastic Syndrome and Solid Tumors
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 Type
Interventional
2. Study Status
Record Verification Date
January 2012
Overall Recruitment Status
Unknown status
Study Start Date
January 2012 (undefined)
Primary Completion Date
December 2013 (Anticipated)
Study Completion Date
March 2014 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Asan Medical Center
4. Oversight
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Leukemia, Myelodysplastic Syndrome, Solid Tumors
Keywords
TBI, Fludarabine, CD3 depletion, Children and adolescents, Haploidentical hematopoietic stem cell transplantation
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
HAPLO
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
anti-thymocyte globulin
Intervention Description
On days -10 to -9
Intervention Type
Biological
Intervention Name(s)
filgrastim
Intervention Description
Beginning on day 4 and continuing until blood counts recover
Intervention Type
Radiation
Intervention Name(s)
Total body irradiation
Intervention Description
2Gy D-6 to D-4
Intervention Type
Drug
Intervention Name(s)
Fludarabine
Intervention Description
30mg/M2 once daily IV on days -8 to -4
Intervention Type
Drug
Intervention Name(s)
cyclophosphamide
Intervention Description
60 mg/kg IV on day-3 and -2
Intervention Type
Drug
Intervention Name(s)
Tacrolimus
Intervention Description
begin on 0
Intervention Type
Drug
Intervention Name(s)
Mycophenolate mofetil
Intervention Description
begin on 0
Intervention Type
Drug
Intervention Name(s)
Rituximab
Intervention Description
375mg/m2 on day +21
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
Engraftment and graft failure rates
Description
Number of patients who failed to stable engraftment by 28 days
Time Frame
28 days engraftment and graft failure
Title
Incidence of acute GVHD
Description
Number of patients with acute GVHD.
Time Frame
100 days post-transplant
Title
Treatment related mortality
Description
Number of death after transplantation
Time Frame
100 days post-transplant
Title
Relapse rate and overall survival
Time Frame
2 year after transplantation
10. Eligibility
Sex
All
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria
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)
No HLA-identical family member or closely matched (8 or 7 of 8 HLA-locus match) unrelated marrow donor available
HLA-haploidentical related donor available
Exclusion criteria
Active fungal infections
HIV positive
Pregnant or nursing
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Ho Joon Im, MD & PhD
Phone
82-2-3010-3371
Email
hojim@amc.seoul.kr
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ho Joon Im, MD & PhD
Organizational Affiliation
Asan Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Asan Medical Center
City
Seoul
ZIP/Postal Code
138-736
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ho Joon Im, MD & PhD
Phone
82-2-3010-3371
Email
hojim@amc.seoul.kr
First Name & Middle Initial & Last Name & Degree
Ho Joon Im, MD & PhD
12. IPD Sharing Statement
Citations:
PubMed Identifier
22055111
Citation
Koh KN, Im HJ, Kim BE, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ. Haploidentical haematopoietic stem cell transplantation using CD3 or CD3/CD19 depletion and conditioning with fludarabine, cyclophosphamide and antithymocyte globulin for acquired severe aplastic anaemia. Br J Haematol. 2012 Apr;157(1):139-42. doi: 10.1111/j.1365-2141.2011.08924.x. Epub 2011 Nov 5. No abstract available.
Results Reference
background
PubMed Identifier
18978746
Citation
Lang P, Handgretinger R. Haploidentical SCT in children: an update and future perspectives. Bone Marrow Transplant. 2008 Oct;42 Suppl 2:S54-9. doi: 10.1038/bmt.2008.285.
Results Reference
background
PubMed Identifier
25310303
Citation
Im HJ, Koh KN, Suh JK, Lee SW, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ. Refinement of treatment strategies in ex vivo T-cell-depleted haploidentical SCT for pediatric patients. Bone Marrow Transplant. 2015 Feb;50(2):225-31. doi: 10.1038/bmt.2014.232. Epub 2014 Oct 13.
Results Reference
derived
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HLA-haploidentical Hematopoietic Stem Cell Transplantation for Children and Adolescents With Acute Leukemia, Myelodysplastic Syndrome and Solid Tumors
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