T-Regulatory Cell Infusion Post Umbilical Cord Blood Transplant in Patients With Advanced Hematologic Cancer

NCT ID: NCT00602693

Last Updated: 2017-12-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

41 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-07-23

Study Completion Date

2015-04-16

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

RATIONALE: Giving chemotherapy, such as fludarabine and cyclophosphamide, and total-body irradiation before a donor umbilical cord blood transplant helps stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Giving an infusion of the donor's T-regulatory cells after the transplant may decrease this effect. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. However, the donor immune system may also react against the recipient's tissues (graft-versus-host disease).

PURPOSE: This phase I trial is studying the side effects and best dose of donor T-regulatory cells after an umbilical cord blood transplant in treating patients with advanced hematologic cancer or other disorder.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

OBJECTIVES:

Primary

* Determine the maximum tolerated dose (MTD) of umbilical cord blood (UCB)-derived T-regulatory (Treg) cells.

Secondary

* Estimate the proportion of patients with detectable circulating Treg cells at 0, 1, 3, 7, and 14 days after infusion.
* Estimate the risk of grades II-IV and III-IV acute graft versus host disease (GVHD) at day +100 with the infusion of Treg cells.
* Estimate the proportion of patients with sustained donor engraftment.
* Estimate the proportion of patients with double chimerism at 6 months and 1 year.
* Determine the speed and cumulative incidence of neutrophil recovery by day 42 and platelet recovery by 6 months after UCB transplantation.
* Estimate the risk of chronic GVHD at 1 year.
* Estimate the probability of disease-free survival at 100 days and 1 year.
* Estimate the risk of fungal and viral infections at 1 year
* Estimate the risk of relapse at 1 year
* Characterize the pattern of immune cell recovery over 1 year

OUTLINE: This is a dose-escalation study of umbilical cord blood (UCB)-derived T-regulatory (Treg) cells. Patients receive nonmyeloablative UCB transplantation and post-transplant immunosuppression as in protocol UMN-2005LS036 (without antithymocyte globulin during conditioning regimen).

* Nonmyeloablative conditioning and UCB transplantation: Patients receive allopurinol on days -7 to day 0, fludarabine phosphate intravenously (IV) over 1 hour on days -6 to -2 and cyclophosphamide IV over 2 hours on day -6; undergo total-body irradiation (TBI) once on day -1; and undergo UCB transplantation on day 0.
* Immunosuppression therapy: Beginning on day -3 and continuing until day +100, patients receive sirolimus intravenously (IV) with 8-12 mg oral loading dose followed by a single dose of 4mg/day with a target serum concentration of 3-12 mg/mL with a taper until day +180. Patients also receive mycophenolate mofetil IV or orally every 8 hours on days -3 to +30.
* Radiation therapy: total body irradiation is administered on Day -1 of 200 cGy.
* UCB Treg cell infusion: Patients receive escalating doses of UCB-derived CD4+ CD25+ Treg cells IV on day +1 (and Day +15 for dose level 5 only) until the maximum tolerated dose is obtained.

After completion of study treatment, patients are followed at day 180, 360, and 720.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Leukemia Lymphoma Multiple Myeloma Plasma Cell Neoplasm Myelodysplastic Syndromes

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

chronic myelogenous leukemia recurrent mantle cell lymphoma prolymphocytic leukemia advanced chronic lymphocytic leukemia small lymphocytic lymphoma B-cell lymphoma follicular lymphoma diffuse large cell lymphoma anaplastic large cell lymphoma Hodgkin lymphoma multiple myeloma

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

UCB post-transplant Treg Cell Infusion

Includes patients with high risk malignancy receiving allopurinol, fludarabine phosphate, cyclophosphamide, sirolimus, total body irradiation, double umbilical cord blood transplantation and Treg infusion cells after transplant. Patients will receive differing dose levels as they are entered and assigned to determine the maximum tolerated dose.

Group Type EXPERIMENTAL

umbilical cord blood transplantation

Intervention Type BIOLOGICAL

Infusion of umbilical cord blood

Allopurinol

Intervention Type DRUG

Administration begins Day -7 through Day 0, tablet or powder prescribed on an individual basis.

fludarabine phosphate

Intervention Type DRUG

40 mg/m\^2 intravenously over 1 hour on Days -6, -5, -4, -3, -2

Cyclophosphamide

Intervention Type DRUG

50 mg/kg intravenous over 2 hours on Day -6

Total body irradiation

Intervention Type RADIATION

200 cGy on Day -1

Treg infusion

Intervention Type BIOLOGICAL

Infusion of T regulatory cells on Day +1 (also Day +15 for Dose level 5 only). Dose escalation ranges include 1, 3, 10, 30, 100, 300 1000, and 300 x 10\^5 Treg/kg.

Sirolimus

Intervention Type DRUG

Beginning on day -3 and continuing until day +100, patients receive sirolimus intravenously (IV) with 8-12 mg oral loading dose followed by a single dose of 4mg/day with a target serum concentration of 3-12 mg/mL with a taper until day +180.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

umbilical cord blood transplantation

Infusion of umbilical cord blood

Intervention Type BIOLOGICAL

Allopurinol

Administration begins Day -7 through Day 0, tablet or powder prescribed on an individual basis.

Intervention Type DRUG

fludarabine phosphate

40 mg/m\^2 intravenously over 1 hour on Days -6, -5, -4, -3, -2

Intervention Type DRUG

Cyclophosphamide

50 mg/kg intravenous over 2 hours on Day -6

Intervention Type DRUG

Total body irradiation

200 cGy on Day -1

Intervention Type RADIATION

Treg infusion

Infusion of T regulatory cells on Day +1 (also Day +15 for Dose level 5 only). Dose escalation ranges include 1, 3, 10, 30, 100, 300 1000, and 300 x 10\^5 Treg/kg.

Intervention Type BIOLOGICAL

Sirolimus

Beginning on day -3 and continuing until day +100, patients receive sirolimus intravenously (IV) with 8-12 mg oral loading dose followed by a single dose of 4mg/day with a target serum concentration of 3-12 mg/mL with a taper until day +180.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

UCB transplant Zyloprim Fludara Cytoxan radiation Treg cells Rapamune®

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Ages 18 to 75 years old
* Eligible for and co-enrolled on protocol UMN-2005LS036, for treatment of any of the following advanced hematologic malignancies:

* Acute leukemias in complete remission (high risk CR1 or subsequent CR); chronic myelogenous leukemia (except refractory blast crisis); myelodysplastic syndrome with severe pancytopenia or complex cytogenetics, large-cell lymphoma, Hodgkin's lymphoma and multiple myeloma, chronic lymphocytic leukemia/small lymphocytic lymphoma, marginal zone b-cell lymphoma, follicular lymphoma, lymphoplasmacytic lymphoma, mantle-cell lymphoma, prolymphocytic leukemia may be eligible after initial therapy.
* Have three partially HLA matched umbilical cord blood (UCB) units (1-2 units for UCB transplantation per MT2005-02 and 1 unit for the Treg cell infusion.)
* Adequate organ function

Exclusion Criteria

* Patients not exposed to highly immunosuppressive single agent or multi-agent chemotherapy within 3 months, or an ablative preparative regimen for autologous hematopoietic stem cell transplant (HCT) within 1 year.
* Pregnancy or breastfeeding
* Current active serious infection
* Evidence of human immunodeficiency virus (HIV) or known HIV positive serology
* Patients with acute leukemia in morphologic relapse/persistent disease defined as \>5% blasts in a \> or = 15% cellular bone marrow or any % blasts if blasts have unique morphologic markers (e.g., Auer rods) or associated cytogenetic markers that allows morphologic relapse to be distinguished are not eligible.
* Chronic myelogenous leukemia in refractory blast crisis.
* Active central nervous system malignancy.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Claudio G. Brunstein, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Masonic Cancer Center, University of Minnesota

Margaret L. MacMillan, MD

Role: PRINCIPAL_INVESTIGATOR

Masonic Cancer Center, University of Minnesota

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Masonic Cancer Center at University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

References

Explore related publications, articles, or registry entries linked to this study.

Brunstein CG, Miller JS, Cao Q, McKenna DH, Hippen KL, Curtsinger J, Defor T, Levine BL, June CH, Rubinstein P, McGlave PB, Blazar BR, Wagner JE. Infusion of ex vivo expanded T regulatory cells in adults transplanted with umbilical cord blood: safety profile and detection kinetics. Blood. 2011 Jan 20;117(3):1061-70. doi: 10.1182/blood-2010-07-293795. Epub 2010 Oct 15.

Reference Type DERIVED
PMID: 20952687 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

MT2006-01

Identifier Type: OTHER

Identifier Source: secondary_id

0701M00303

Identifier Type: OTHER

Identifier Source: secondary_id

2007LS022

Identifier Type: -

Identifier Source: org_study_id