Status:

TERMINATED

Combined T Cell Depleted Haploidentical Peripheral Blood Stem Cell and Unrelated Umbilical Cord Blood Transplantation in Patients With Hematologic Malignancies Using a Total Lymphoid Irradiation Based Preparative Regimen

Lead Sponsor:

St. Jude Children's Research Hospital

Conditions:

Hematological Malignancies

Eligibility:

All Genders

Up to 21 years

Phase:

PHASE2

Brief Summary

In this study, participants with high-risk hematologic malignancies undergoing hematopoietic cell transplantation (HCT), who do not have a suitable human leukocyte antigen (HLA)-matched related/siblin...

Detailed Description

Prior to stem cell infusion, participants will receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. T...

Eligibility Criteria

Inclusion

  • Inclusion Criteria-Transplant Recipient:
  • Age less than or equal to 21 years old.
  • Does not have a suitable matched related/sibling donor (MSD) or volunteer matched unrelated donor (MUD) available in the necessary time for stem cell donation.
  • Has a suitable partially human leukocyte antigen (HLA)-matched (≥ 3 of 6) family member donor.
  • Has a partially HLA-matched single umbilical cord blood (UCB) unit (≥ 4 of 6) with adequate cell dose. UCB units must fulfill eligibility as outlined in 21 CFR 1271 and agency guidance.
  • High-risk hematologic malignancy.
  • High risk acute lymphocytic leukemia (ALL) in complete remission-1 (CR)1. \[Examples include, but not limited to t(9;22), hypodiploid,, M2 or greater marrow at the end of induction, infants with mixed lineage leukemia (MLL) fusion or t(4;11)\].
  • ALL in High risk CR2. \[Examples include but not limited to t(9;22), bone marrow (BM) relapse \<36 mo CR1, T-ALL, very early (\< 6mo CR1) isolated central nervous system (CNS) relapse.\]
  • ALL in CR3 or subsequent.
  • Acute myeloid leukemia (AML) in high risk CR1. \[Examples include but not limited to preceding MDS, 5q-, -5, -7, FAB M6, FAB M7 not t(1;22), minimal residual disease (MRD) ≥ 5% on day 22 (AML08), M3 marrow after induction 1, M2 marrow after two cycles of induction, FLT3-ITD.\]
  • AML in CR2 or subsequent.
  • Therapy related AML, with prior malignancy in CR \> 12mo
  • Myelodysplastic syndrome (MDS), primary or secondary
  • Natural killer (NK) cell, biphenotypic, or undifferentiated leukemia in CR1 or subsequent.
  • Chronic myeloid leukemia (CML) in accelerated phase, or in chronic phase with persistent molecular positivity or intolerance to tyrosine kinase inhibitor.
  • Hodgkin lymphoma in CR2 or subsequent after failure of prior autologous hematopoietic cell transplantation (HCT), or unable to mobilize stem cells for autologous HCT.
  • Non-Hodgkin lymphoma in CR2 or subsequent.
  • Juvenile myelomonocytic leukemia (JMML).
  • Refractory hematologic malignancies \[ALL, AML, chronic myeloid leukemia (CML) in blast crisis, Hodgkin or non-Hodgkin lymphoma\] due to chemoresistant relapse or primary induction failure.
  • All patients with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for study.
  • Patient must fulfill pre-transplant evaluation:
  • Cardiac Function: Left ventricular ejection fraction (LVEF) ≥ 40% or shortening fraction (SF) ≥ 25%.
  • Creatinine clearance (CrCL) or glomerular filtration rate (GFR) ≥ 50 ml/min/1.73m2.
  • Forced vital capacity (FVC) ≥ 50% of predicted value or pulse oximetry (Pox) ≥ 92% on room air.
  • Karnofsky or Lansky performance score ≥ 50.
  • Bilirubin ≤ 3 times the upper limit of normal for age.
  • Alanine aminotransferase (ALT) ≤ 5x the upper limit of normal for age.
  • Aspartate aminotransferase (AST) ≤ 5x the upper limit of normal for age.
  • Exclusion Criteria - Transplant Recipient:
  • Patient has a suitable MSD, volunteer matched unrelated donor (MURD), or killer-immunoglobulin receptors (KIR) mismatched haploidentical donor available in the necessary time for stem cell donation.
  • Patient has any other active malignancy other than the one for which HCT is indicated.
  • Patient is pregnant as confirmed by positive serum or urine pregnancy test within 14 days prior to enrollment.
  • Patient is breast feeding.
  • Patient has Down Syndrome.
  • Patient has a current uncontrolled bacterial, fungal, or viral infection per the judgment of the principal investigator.
  • Inclusion criteria - haploidentical donor
  • At least single haplotype matched (≥ 3 of 6) family member
  • At least 18 years of age.
  • Human immunodeficiency virus (HIV) negative.
  • Not pregnant as confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment (if female).
  • Not breast feeding.
  • Regarding eligibility, is identified as either:
  • Completed the process of donor eligibility determination as outlined in 21 CFR 1271 and agency guidance; OR
  • Does not meet 21 CFR 1271 eligibility requirements, but has a declaration of urgent medical need completed by the principal investigator or physician sub-investigator per 21 CFR 1271.

Exclusion

    Key Trial Info

    Start Date :

    July 11 2014

    Trial Type :

    INTERVENTIONAL

    Allocation :

    ACTUAL

    End Date :

    May 23 2017

    Estimated Enrollment :

    24 Patients enrolled

    Trial Details

    Trial ID

    NCT02199041

    Start Date

    July 11 2014

    End Date

    May 23 2017

    Last Update

    February 7 2018

    Active Locations (1)

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    St. Jude Children's Research Hospital

    Memphis, Tennessee, United States, 38105

    Combined T Cell Depleted Haploidentical Peripheral Blood Stem Cell and Unrelated Umbilical Cord Blood Transplantation in Patients With Hematologic Malignancies Using a Total Lymphoid Irradiation Based Preparative Regimen | DecenTrialz