Clinical trial NCT01509300: 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
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Acute Leukemia
Myelodysplastic Syndrome
Solid Tumors
Keywords: TBI
Fludarabine
CD3 depletion
Children and adolescents
Haploidentical hematopoietic stem cell transplantation
Lead sponsor: Asan Medical Center
Sponsor class: OTHER
First posted: 2012-01-13
Enrollment: 10
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Transplantation-related mortality and overall survival of TBI, Fludarabine, Cyclophosphamide and anti-thymocyte globulin for engraftment of CD3 depleted haploidentical peripheral blood stem cells.
timeFrame: 2 years post-transplant
Secondary outcomes:
measure: Engraftment and graft failure rates
timeFrame: 28 days engraftment and graft failure
measure: Incidence of acute GVHD
timeFrame: 100 days post-transplant
measure: Treatment related mortality
timeFrame: 100 days post-transplant
measure: Relapse rate and overall survival
timeFrame: 2 year after transplantation
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
Study officials:
affiliation: Asan Medical Center
name: Ho Joon Im, MD & PhD
role: PRINCIPAL_INVESTIGATOR
References:
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.
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.
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.