NCT00376519

Brief Summary

RATIONALE: Giving chemotherapy, such as fludarabine and cyclophosphamide, and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer or abnormal cells and prepares the patient's bone marrow for the stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer or abnormal cells (graft-versus-tumor effect). Giving an infusion of the donor's T-regulatory cells before the transplant may help increase this effect. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening. PURPOSE: This phase I trial is studying the side effects and best dose of umbilical cord blood T-regulatory cell infusion followed by donor umbilical cord blood transplant in treating patients with high-risk leukemia or other hematologic diseases.

Trial Health

57
Monitor

Trial Health Score

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

Enrollment
3

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started May 2007

Geographic Reach
1 country

1 active site

Status
terminated

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

First Submitted

Initial submission to the registry

September 13, 2006

Completed
2 days until next milestone

First Posted

Study publicly available on registry

September 15, 2006

Completed
8 months until next milestone

Study Start

First participant enrolled

May 1, 2007

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2008

Completed
Last Updated

December 2, 2017

Status Verified

November 1, 2017

Enrollment Period

10 months

First QC Date

September 13, 2006

Last Update Submit

November 29, 2017

Conditions

Keywords

graft versus host diseaseadult acute lymphoblastic leukemia in remissionadult acute myeloid leukemia in remissionsecondary acute myeloid leukemiarefractory anemia with excess blastsmyelodysplastic syndromeschronic myelogenous leukemiarefractory anemiaprolymphocytic leukemiastage IV adult lymphoblastic lymphomarecurrent adult Burkitt lymphomastage I adult Burkitt lymphomastage III adult Burkitt lymphomastage IV adult Burkitt lymphomafollicular lymphomamantle cell lymphomachronic idiopathic myelofibrosissecondary myelofibrosisrefractory chronic lymphocytic leukemiarecurrent small lymphocytic lymphomarecurrent marginal zone lymphomamarginal zone lymphomadiffuse large cell lymphomaanaplastic large cell lymphomalymphoblastic lymphomaimmunoblastic large cell lymphomamultiple myeloma

Outcome Measures

Primary Outcomes (1)

  • Maximum tolerated dose of T-regulatory cells

    Dose limiting toxicities (DLT) are defined as any grade 3-4 toxicity within 24 hours of Treg cell infusion, excluding hematological .

    Within 24 Hours

Secondary Outcomes (6)

  • Incidence of neutrophil recovery

    Day 42

  • Incidence of double chimerism

    Day 21

  • Incidence of grades II-IV and III-IV acute graft-versus-host disease (GVHD)

    Day 100

  • Incidence of chronic graft-versus-host disease

    1 Year

  • Survival

    Day 100 and 1 Year

  • +1 more secondary outcomes

Study Arms (1)

Transplant with Treg Cells

EXPERIMENTAL

Patients receive preparative therapy with Fludarabine, cyclophosphamide, total body irradiation and Treg infusion followed by umbilical cord blood transplantation.

Drug: cyclophosphamideDrug: cyclosporineDrug: fludarabine phosphateDrug: mycophenolate mofetilProcedure: Treg cell infusionProcedure: umbilical cord blood transplantationRadiation: total-body irradiation

Interventions

Cyclophosphamide 60 mg/kg/day will be administered as a 2 hour intravenous infusion with a high volume fluid flush and mesna (MT(S)9006) on day -8 and -7 one hour after fludarabine infusion.

Also known as: Cytoxan
Transplant with Treg Cells

Will be administered beginning on day -3 and adjusted to maintain a level of \>200. For adults the initial dose will be 2.5 mg/kg intravenously (IV) over 2 hours every 12 hours.

Also known as: CSA
Transplant with Treg Cells

Fludarabine 25 mg/m\^2/day will be administered as a 1 hour intravenous infusion on days -9 through -7.

Also known as: Fludara
Transplant with Treg Cells

All patients will begin mycophenolate mofetil (MMF) on day -3, at the dose of 3 grams/day divided into 2 or 3 doses (every 12 or 8 hours).

Also known as: MMF
Transplant with Treg Cells

On day -1 prior to UCB transplantation, Treg cells will be infused IV without in-line filtration. A semi-log dose escalation of CD4+CD25+ Treg cells is scheduled with 3 patients at each step. Doses will be 0.1 x 10\^6/kg, 0.3 x 10\^6/kg, 1 x 10\^6/kg and 3 x 10\^6/kg weight (determined on the day prior to administration of the preparative therapy).

Transplant with Treg Cells

Following the administration of the preparative therapy, all subjects will undergo UCB transplantation. The UCB will be administrated by IV infusion without in-line filtration.

Also known as: UCBT
Transplant with Treg Cells

Radiotherapy: 165 cGy will be administered on day -5 through -2 two fractions each day.

Transplant with Treg Cells

Eligibility Criteria

Age18 Years - 45 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • Patient and Donor Demographic Criteria
  • Patient must be 18-45 years of age.
  • Patients must have three partially HLA matched UCB units. Units identified as the HSC source must be HLA matched at 4-6 HLA- A and B (at low to intermediate resolution) and DRB1 (at high resolution), and the units must be HLA matched at 4-6 HLA- A, B, DRB1 antigens with each other. Total cryopreserved HSC graft cell dose must be \>2.5 x 107 nucleated cells per kilogram recipient body weight. Also, the two umbilical cord blood (UCB) units must be ABO-matched.
  • The UCB unit identified as the Treg source must be HLA matched at 4-6 HLA antigens with the patient (without an HLA or ABO matching criterion with the UCB HSC source).
  • Disease Criteria
  • Patients must have a hematological malignancy as listed below:
  • Acute myelogenous leukemia: high risk CR1 (as evidenced by preceding myelodysplastic syndrome (MDS), high risk cytogenetics such as those associated with MDS or complex karyotype, or \>2 cycles to obtain complete remission (CR); second or greater CR. Must be in remission by morphology (\<5% blasts within normocellular marrow).
  • Acute lymphocytic leukemia: high risk CR1 as evidenced by high risk cytogenetics \[t(9;22), t (1:19), t(4;11) or other MLL rearrangements\] or \> 1 cycle to obtain CR; second or greater CR.
  • Chronic myelogenous leukemia resistant to imatinib therapy
  • Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt) or refractory anemia with severe pancytopenia or high risk cytogenetics. Blasts must be \< 10% by a representative bone marrow aspirate morphology (otherwise induction chemotherapy to achieve \< 10% blasts is required pre-transplant).
  • Advanced myelofibrosis
  • Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone B-cell lymphoma or follicular lymphoma that have progressed after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for debulking chemotherapy before transplant.
  • Lymphoplasmacytic lymphoma, mantle-cell lymphoma, prolymphocytic leukemia are eligible after initial therapy in CR1+ or PR1+.
  • Large cell non-Hodgkins lymphoma (NHL) \> CR2/\> PR2. Patients in CR2/PR2 with initial short remission(\<6 months) are eligible.
  • Lymphoblastic lymphoma, Burkitt's lymphoma, and other high-grade NHL after initial therapy if stage III/IV in CR1/PR1 or after progression if stage I/II \<1 year.
  • +3 more criteria

You may not qualify if:

  • Pregnant or breastfeeding
  • Evidence of HIV infection or known HIV positive serology
  • Current active infection
  • Available HLA matched sibling donor.
  • CML in active blast crisis

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Masonic Cancer Center at University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Graft vs Host DiseaseLeukemiaLymphomaMultiple MyelomaNeoplasms, Plasma CellMyelodysplastic SyndromesAnemia, Refractory, with Excess of BlastsLeukemia, Myelogenous, Chronic, BCR-ABL PositiveAnemia, RefractoryLeukemia, ProlymphocyticPrecursor Cell Lymphoblastic Leukemia-LymphomaBurkitt LymphomaLymphoma, FollicularLymphoma, Mantle-CellPrimary MyelofibrosisLeukemia, Lymphocytic, Chronic, B-CellLymphoma, B-Cell, Marginal ZoneLymphoma, Large B-Cell, DiffuseLymphoma, Large-Cell, AnaplasticLymphoma, Large-Cell, Immunoblastic

Interventions

CyclophosphamideCyclosporinefludarabine phosphateMycophenolic AcidCord Blood Stem Cell TransplantationWhole-Body Irradiation

Condition Hierarchy (Ancestors)

Immune System DiseasesNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersHemostatic DisordersVascular DiseasesCardiovascular DiseasesParaproteinemiasBlood Protein DisordersHemorrhagic DisordersBone Marrow DiseasesAnemiaLeukemia, MyeloidMyeloproliferative DisordersChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsLeukemia, LymphoidEpstein-Barr Virus InfectionsHerpesviridae InfectionsDNA Virus InfectionsVirus DiseasesInfectionsTumor Virus InfectionsLymphoma, B-CellLymphoma, Non-HodgkinLeukemia, B-CellLymphoma, T-Cell

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus CompoundsCyclosporinsPeptides, CyclicMacrocyclic CompoundsPolycyclic CompoundsPeptidesAmino Acids, Peptides, and ProteinsCaproatesAcids, AcyclicCarboxylic AcidsFatty AcidsLipidsStem Cell TransplantationCell TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, OperativeRadiotherapyInvestigative Techniques

Study Officials

  • Claudio G. Brunstein, MD, PhD

    Masonic Cancer Center, University of Minnesota

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 13, 2006

First Posted

September 15, 2006

Study Start

May 1, 2007

Primary Completion

March 1, 2008

Study Completion

March 1, 2008

Last Updated

December 2, 2017

Record last verified: 2017-11

Locations