NCT02727803

Brief Summary

This phase II clinical trial studies how well personalized natural killer (NK) cell therapy works after chemotherapy and umbilical cord blood transplant in treating patients with myelodysplastic syndrome, leukemia, lymphoma or multiple myeloma. This clinical trial will test cord blood (CB) selection for human leukocyte antigen (HLA)-C1/x recipients based on HLA-killer-cell immunoglobulin-like receptor (KIR) typing, and adoptive therapy with CB-derived NK cells for HLA-C2/C2 patients. Natural killer cells may kill tumor cells that remain in the body after chemotherapy treatment and lessen the risk of graft versus host disease after cord blood transplant.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
100

participants targeted

Target at P50-P75 for phase_2

Timeline
13mo left

Started May 2016

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress90%
May 2016May 2027

First Submitted

Initial submission to the registry

March 25, 2016

Completed
11 days until next milestone

First Posted

Study publicly available on registry

April 5, 2016

Completed
1 month until next milestone

Study Start

First participant enrolled

May 19, 2016

Completed
11 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 31, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 31, 2027

Last Updated

May 5, 2026

Status Verified

November 1, 2025

Enrollment Period

11 years

First QC Date

March 25, 2016

Last Update Submit

April 29, 2026

Conditions

Outcome Measures

Primary Outcomes (2)

  • Progression free survival (PFS) time in C2C2 patients

    Distributions of time-to-event variables will be estimated using the method of Kaplan and Meier, and Bayesian regression models will be used to assess the relationship of each outcome with patient covariates, including disease stage, KIR haplotype, age, diagnosis, human leukocyte antigen (HLA) match, cytomegalovirus (CMV) status, and gender. Categorical outcomes will be evaluated by tabulation and Bayesian regression modeling.

    From the date of engraftment to disease progression or death, assessed up to 4 years

  • Progression free survival (PFS) time in C1 patients

    Distributions of time-to-event variables will be estimated using the method of Kaplan and Meier, and Bayesian regression models will be used to assess the relationship of each outcome with patient covariates, including disease stage, KIR haplotype, age, diagnosis, HLA match, CMV status, and gender. Categorical outcomes will be evaluated by tabulation and Bayesian regression modeling.

    From the date of cord blood transplant to disease progression or death, assessed up to 4 years

Secondary Outcomes (4)

  • Overall survival time

    Up to 4 years

  • Incidence of transplant related mortality

    Up to 4 years

  • Incidence of graft-versus host disease

    Up to 4 years

  • Incidence of infection

    Up to 4 years

Study Arms (3)

Myeloablative regimen 1

EXPERIMENTAL

Patients receive anti-thymocyte globulin IV over 4 hours on days -9 and -8, fludarabine phosphate IV over 1 hour, clofarabine IV over 1 hour, and busulfan IV over 3 hours on days -7 to -4. Patients undergo TBI on day -3. UMBILICAL CORD BLOOD TRANSPLANT: Patients undergo umbilical cord blood transplantation on day 0. NK CELLS INFUSION: Patients receive NK cells IV over 30 minutes between days 30-180.

Biological: Allogeneic Natural Killer Cell Line NK-92Biological: Anti-Thymocyte GlobulinDrug: BusulfanDrug: ClofarabineDrug: Fludarabine PhosphateOther: Laboratory Biomarker AnalysisRadiation: Total-Body IrradiationProcedure: Umbilical Cord Blood Transplantation

Non-myeloablative regimen 2

EXPERIMENTAL

Patients with CD20 positive malignancies receive rituximab IV over 6 hours on day -9. Patients receive anti-thymocyte globulin IV over 4 hours on days -8 and -7, fludarabine phosphate IV over 1 hour on days -6 to -3, and cyclophosphamide IV over 3 hours on day -6 and undergo TBI on day -1 at the discretion of the investigator(s). UMBILICAL CORD BLOOD TRANSPLANT: Patients undergo umbilical cord blood transplantation on day 0. NK CELLS INFUSION: Patients receive NK cells IV over 30 minutes between days 30-180.

Biological: Allogeneic Natural Killer Cell Line NK-92Biological: Anti-Thymocyte GlobulinDrug: CyclophosphamideDrug: Fludarabine PhosphateOther: Laboratory Biomarker AnalysisBiological: RituximabRadiation: Total-Body IrradiationProcedure: Umbilical Cord Blood Transplantation

Reduced intensity regimen 3

EXPERIMENTAL

Patients receive anti-thymocyte globulin IV over 4 hours on days -7 and -6, fludarabine phosphate IV over 1 hour on days -5 to -2, and melphalan IV over 30 minutes on day -2. UMBILICAL CORD BLOOD TRANSPLANT: Patients undergo umbilical cord blood transplantation on day 0. NK CELLS INFUSION: Patients receive NK cells IV over 30 minutes between days 30-180.

Biological: Allogeneic Natural Killer Cell Line NK-92Biological: Anti-Thymocyte GlobulinDrug: Fludarabine PhosphateOther: Laboratory Biomarker AnalysisDrug: MelphalanProcedure: Umbilical Cord Blood Transplantation

Interventions

Given IV

Also known as: haNK, NK-92, NK-92 Cells
Myeloablative regimen 1Non-myeloablative regimen 2Reduced intensity regimen 3

Given IV

Also known as: Antithymocyte Globulin, Antithymocyte Serum, ATG, ATS
Myeloablative regimen 1Non-myeloablative regimen 2Reduced intensity regimen 3

Given IV

Also known as: 1, 4-Bis[methanesulfonoxy]butane, BUS, Bussulfam, Busulfanum, Busulfex, Busulphan, CB 2041, CB-2041, Glyzophrol, GT 41, GT-41, Joacamine, Methanesulfonic Acid Tetramethylene Ester, Methanesulfonic acid, tetramethylene ester, Mielucin, Misulban, Misulfan, Mitosan, Myeleukon, Myeloleukon, Myelosan, Mylecytan, Myleran, Sulfabutin, Tetramethylene Bis(methanesulfonate), Tetramethylene bis[methanesulfonate], WR-19508
Myeloablative regimen 1

Given IV

Also known as: Clofarex, Clolar
Myeloablative regimen 1

Given IV

Also known as: (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida, Ciclofosfamide, Cicloxal, Clafen, Claphene, CP monohydrate, CTX, CYCLO-cell, Cycloblastin, Cycloblastine, Cyclophospham, Cyclophosphamid monohydrate, Cyclophosphamidum, Cyclophosphan, Cyclophosphane, Cyclophosphanum, Cyclostin, Cyclostine, Cytophosphan, Cytophosphane, Cytoxan, Fosfaseron, Genoxal, Genuxal, Ledoxina, Mitoxan, Neosar, Revimmune, Syklofosfamid, WR- 138719
Non-myeloablative regimen 2

Given IV

Also known as: 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, Fludara, SH T 586
Myeloablative regimen 1Non-myeloablative regimen 2Reduced intensity regimen 3

Correlative studies

Myeloablative regimen 1Non-myeloablative regimen 2Reduced intensity regimen 3

Given IV

Also known as: Alanine Nitrogen Mustard, CB-3025, L-PAM, L-Phenylalanine Mustard, L-sarcolysin, L-Sarcolysin Phenylalanine mustard, L-Sarcolysine, Melphalanum, Phenylalanine Mustard, Phenylalanine nitrogen mustard, Sarcoclorin, Sarkolysin, WR-19813
Reduced intensity regimen 3
RituximabBIOLOGICAL

Given IV

Also known as: ABP 798, BI 695500, C2B8 Monoclonal Antibody, Chimeric Anti-CD20 Antibody, CT-P10, IDEC-102, IDEC-C2B8, IDEC-C2B8 Monoclonal Antibody, MabThera, Monoclonal Antibody IDEC-C2B8, PF-05280586, Rituxan, Rituximab Biosimilar ABP 798, Rituximab Biosimilar BI 695500, Rituximab Biosimilar CT-P10, Rituximab Biosimilar GB241, Rituximab Biosimilar IBI301, Rituximab Biosimilar PF-05280586, Rituximab Biosimilar RTXM83, Rituximab Biosimilar SAIT101, rituximab biosimilar TQB2303, rituximab-abbs, RTXM83, Truxima
Non-myeloablative regimen 2

Undergo total body irradiation

Also known as: Total Body Irradiation, Whole-Body Irradiation
Myeloablative regimen 1Non-myeloablative regimen 2

Undergo umbilical cord blood transplantation

Also known as: Cord Blood Transplantation, UCB transplantation
Myeloablative regimen 1Non-myeloablative regimen 2Reduced intensity regimen 3

Eligibility Criteria

Age15 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patients must have one of the following hematologic malignancies: acute myelogenous leukemia (AML), induction failure, high-risk for relapse first remission (with intermediate-risk or high-risk cytogenetics including complex karyotype, abnormal \[abn\]\[3q\], -5/5q-, -7/7q-, abn\[12p\], abn\[17p\], myeloid/lymphoid or mixed-lineage leukemia \[MLL\] gene re-arrangement and t \[6;9\]47, fms related tyrosine kinase 3 \[flt3\] mutation positive and/or evidence of minimal residual disease by flow cytometry), secondary leukemia from prior chemotherapy and/or arising from myelodysplastic syndromes (MDS), any disease beyond first remission
  • Myelodysplastic syndrome (MDS): Primary or therapy related, including patients that will be considered for transplant; these include any of the following categories: 1) revised International Prognostic Scoring System (IPSS) intermediate and high risk groups, 2) malondialdehyde (MDA) with transfusion dependency, 3) failure to respond or progression of disease on hypomethylating agents, 4) refractory anemia with excess of blasts, 5) transformation to acute leukemia, 6) chronic myelomonocytic leukemia, 7) atypical MDS/myeloproliferative syndromes, 8) complex karyotype, abn(3g), -5/5g-, -7/7g-, abn(12p), abn(17p)
  • Acute lymphoblastic leukemia (ALL): Induction failure, primary refractory to treatment (do not achieve complete remission after first course of therapy) or are beyond first remission including second or greater remission or active disease; patients in first remission are eligible if they are considered high risk, defined as any of the following detected at any time: with translocations 9;22 or 4;11, hypodiploidy, complex karyotype, secondary leukemia developing after cytotoxic drug exposure, and/or evidence of minimal residual disease or acute biphenotypic leukemia, or double hit non-Hodgkin's lymphoma
  • Non-Hodgkin's lymphoma (NHL) in second or third complete remission, or relapse (including relapse post autologous hematopoietic stem cell transplant); relapsed double hit lymphomas; patients with options for treatment that are known to be curative are not eligible
  • Small lymphocytic lymphoma (SLL), or chronic lymphocytic leukemia (CLL) with progressive disease following a minimum of two lines of standard therapy
  • Chronic myeloid leukemia (CML) second chronic phase or accelerated phase
  • Hodgkin's disease (HD): Induction failures, after first complete remission, or relapse (including relapse post autologous hematopoietic stem cell transplant), or those with active disease
  • Multiple myeloma: stage II or III, symptomatic, secretory multiple myeloma requiring treatment
  • A person (such as a haploidentical family member) or unit of cord blood must be identified as a source of back-up cells source in case of engraftment failure
  • Patient age criteria: age \>= 15 and =\< 45 years (myeloablative regimen 1; age \>= 15 and =\< 80 years (nonmyeloablative regimen 2) at the discretion of the investigator(s); age \>= 15 and =\< 80 years old that in the opinion of the investigator(s) would preclude myeloablative therapy may receive reduced intensity regimen 3
  • Performance score of at least 60% by Karnofsky
  • Left ventricular ejection fraction of at least 40% (myeloablative regimen 1, reduced intensity regimen 3)
  • Left ventricular ejection fraction of at least 30% (nonmyeloablative regimen 2)
  • Pulmonary function test (PFT) demonstrating an adjusted diffusion capacity of least 50% predicted value for hemoglobin concentration (myeloablative regimen 1, reduced intensity regimen 3)
  • Serum creatinine within normal range, or if serum creatinine outside normal range, then renal function (measured or estimated creatinine clearance or glomerular filtration rate \[GFR\]) \> 40mL/min/1.73 m\^2
  • +4 more criteria

You may not qualify if:

  • Human immunodeficiency virus (HIV) positive; HIV results will be determined by nucleic acid testing
  • Uncontrolled serious medical condition such as persistent septicemia despite adequate antibiotic therapy, decompensated congestive heart failure despite cardiac medications or pulmonary insufficiency requiring intubation (excluding primary disease for which cord blood \[CB\] transplantation is proposed), or psychiatric condition that would limit informed consent
  • Active central nervous system (CNS) disease in patient with history of CNS malignancy
  • Availability of appropriate, willing, human leukocyte antigen (HLA)-matched related stem cell donor

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Related Links

MeSH Terms

Conditions

Leukemia, Myeloid, Accelerated PhaseLeukemia, Biphenotypic, AcutePrecursor Cell Lymphoblastic Leukemia-LymphomaLeukemia, Myelomonocytic, ChronicLeukemia, Myeloid, Chronic-PhaseMyelodysplastic SyndromesAnemia, Refractory, with Excess of BlastsMyelodysplastic-Myeloproliferative DiseasesLeukemia, Myeloid, AcuteHodgkin DiseaseLymphoma, Non-Hodgkin

Interventions

Antilymphocyte SerumBusulfanClofarabineCyclophosphamidefludarabine phosphateMelphalanRituximabCT-P10Whole-Body IrradiationCord Blood Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Leukemia, Myelogenous, Chronic, BCR-ABL PositiveLeukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsMyeloproliferative DisordersBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsLeukemia, LymphoidLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesAnemia, RefractoryAnemiaLymphoma

Intervention Hierarchy (Ancestors)

Immune SeraAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsBiological ProductsComplex MixturesButylene GlycolsGlycolsAlcoholsOrganic ChemicalsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicHydrocarbonsSulfonic AcidsSulfur AcidsSulfur CompoundsAdenine NucleotidesPurine NucleotidesPurinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic CompoundsArabinonucleosidesNucleosidesNucleic Acids, Nucleotides, and NucleosidesNucleotidesRibonucleotidesPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedPhosphoramidesOrganophosphorus CompoundsPhenylalanineAmino Acids, AromaticAmino Acids, CyclicAmino AcidsAntibodies, Monoclonal, Murine-DerivedAntibodies, MonoclonalRadiotherapyTherapeuticsInvestigative TechniquesStem Cell TransplantationCell TransplantationCell- and Tissue-Based TherapyBiological TherapyTransplantationSurgical Procedures, Operative

Study Officials

  • Amanda Olson, MD

    M.D. Anderson Cancer Center

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Amanda Olson, MD

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 25, 2016

First Posted

April 5, 2016

Study Start

May 19, 2016

Primary Completion (Estimated)

May 31, 2027

Study Completion (Estimated)

May 31, 2027

Last Updated

May 5, 2026

Record last verified: 2025-11

Locations