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

Asan Medical Center

Status and phase: Unknown; Phase 2; Phase 1

Conditions: Acute Leukemia; Solid Tumors; Myelodysplastic Syndrome

Treatments: Drug: Rituximab; Drug: cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate mofetil; Biological: filgrastim; Biological: anti-thymocyte globulin; Drug: Fludarabine; Radiation: Total body irradiation

Study type: Interventional

Funder types: Other

Identifiers

NCT01509300

AMCPHO-SCT0902

Details and patient eligibility

About

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.

Enrollment: 10 estimated patients

Sex: All

Ages: Under 21 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

HAPLO

Experimental group

Treatment:

Drug: Rituximab

Drug: Tacrolimus

Biological: filgrastim

Biological: anti-thymocyte globulin

Drug: Mycophenolate mofetil

Radiation: Total body irradiation

Drug: cyclophosphamide

Drug: Fludarabine