Engineered Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies | Aplastic Anemia & MDS International Foundation
Engineered Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Clinical Trial: NCT02960646

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Patients receive melphalan IV over 30 minutes on day -6 and fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo total-body irradiation (TBI) on day -2 and CD45RA depleted peripheral blood stem cell transplantation on day 0. Patients also receive cyclophosphamide IV over 3 hours on days 3-4. Beginning on day 5, patients receive tacrolimus IV for 2 weeks and PO for at least 4 months. Beginning on day 7, patients receive filgrastim SC daily. Patients with CD20 positive lymphoma may receive rituximab IV on days -13, -6, 1, and 8.

Study Date: 
Wed, 01/18/2017 to Fri, 02/28/2020
Bone Marrow Disease(s): 
  • acute myeloid leukemia (AML)
  • aplastic anemia
Lack of a human leukocyte antigen (HLA) matched related donor, lack of an immediately available 8/8 HLA matched unrelated donor Patients must be diagnosed with a high-risk and/or advanced hematologic malignancy defined as one of the following Acute lymphocytic leukemia (ALL) in complete remission (CR)1 with high-risk features including adverse cytogenetic such as t(9;22), t(1;19), t(4;11), or MLL gene rearrangements; in second or greater morphologic remission; persistent minimal residual disease Acute myeloid leukemia (AML) in CR1 with intermediate-risk disease and persistent detectable minimal residual disease (MRD), or with high-risk features defined as: greater than 1 cycle of induction therapy required to achieve remission; preceding myelodysplastic syndrome (MDS) or myeloproliferative disease; presence of FLT3 mutations or internal tandem duplications, DNMT3a, TET2, MLL-partial tandem duplication (PTD), ASXL1, PHF6; FAB M6 or M7 classification; adverse cytogenetics including: -5, del 5q, -7, del7q, abnormalities involving 3q, 9q, 11q, 20q, 21q, 17, +8, complex (> 3 abnormalities) Patients with AML must have less than 10% bone marrow blasts and < 100/mcL absolute peripheral blood blast count Patients with AML in CR2, subsequent CR or with active disease at transplant (< 10% bone marrow blasts) MDS with International Prognostic Scoring System (IPSS) intermediate-2 or higher, therapy-related MDS or chronic myelomonocytic leukemia (CMML) Aplastic anemia with absolute neutrophil count (ANC) < 1,000 and transfusion dependent after failed immunosuppression therapy Chronic myeloid leukemia (CML) >= 1st chronic phase, after failed >=2 lines of tyrosine kinase inhibitors; patients who progressed to blast phase must be in morphologic remission at transplant Relapsed Hodgkin's disease or non-Hodgkin's lymphoma (NHL) Patients with chemo-sensitive chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) with persistent or recurrent disease after fludarabine-based regimens with < 25% involvement by CLL/SLL cells Patients with lymphoblastic lymphoma in remission or after partial response to chemotherapy Patients with poor prognosis multiple myeloma by cytogenetics del13, del 17p, t(4;14) or t(14;16) or hypodiploidy, with advanced disease (stage >= 2) and /or relapsed after autologous stem cell transplant Zubrod performance status 0-1 or Karnofsky performance status > 70%; patients > 50 years will have to have a Sorror Comorbidity Index =< 3 Available haploidentical donor willing and eligible to undergo a peripheral blood collection Left ventricular ejection fraction (LVEF) > 40% Bilirubin =< 1.5 mg/dl (unless Gilbert's syndrome), alanine aminotransferase (ALT) or aspartate aminotransferase (AST) =< 200 IU/ml for adults; conjugated (direct) bilirubin < 2 x upper limit of normal Serum creatinine clearance >= 50 ml/min (calculated with Cockcroft-Gault formula) Diffusing capacity for carbon monoxide (DLCO) >= 45% predicted corrected for hemoglobin Patient or patient's legal representative must provide written informed consent