CClinicalTrials.gg
TerminatedNCT01044745Updated Sep 6, 2023Results posted

Rituximab in Preventing Acute Graft-Versus-Host Disease in a Donor Stem Cell Transplant for Hematologic Cancer

A Phase 2 interventional study of rituximab and mycophenolate mofetil in Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission and Adult Acute Myeloid Leukemia in Remission, sponsored by University of Nebraska. Terminated at 1 site in United States. Open to participants aged 19 Years to 75 Years. Per ClinicalTrials.gov, last updated 2023-09-06.

Sponsored by University of Nebraska · Phase 2, Interventional, and Treatment

Why this study was terminated
Study was closed to accrual for safety related to the frequency of BK infections.
Phase
Phase 2
Study type
Interventional
Enrollment
20
Allocation
Not applicable
Ages
19 Years to 75 Years
Sex
All
01

Study summary

This phase II trial is studying how well rituximab works in preventing acute graft-versus-host disease (GVHD) in patients undergoing a donor stem cell transplant for hematologic cancer. Giving chemotherapy and total-body irradiation before a donor stem cell transplant helps stop the growth of cancer cells. It may also 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). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving a monoclonal antibody, rituximab, together with anti-thymocyte globulin, tacrolimus, and mycophenolate mofetil before and after the transplant may stop this from happening

Read the detailed description

PRIMARY OBJECTIVES:

I. To determine the incidence of grade II-IV acute GVHD at day 100 after matched unrelated donor allogeneic hematopoietic cell transplantation (HCT) when incorporating rituximab in the conditioning regimen.

SECONDARY OBJECTIVES:

I. To determine the day 100 transplant related mortality after matched unrelated donor allogeneic HCT when incorporating rituximab in the conditioning regimen.

II. To determine overall survival (OS) and disease-free survival (DFS) after matched unrelated donor allogeneic HCT when incorporating rituximab in the conditioning regimen.

III. To determine the cumulative incidence of infectious complications at day 100 after matched unrelated donor HCT when incorporating rituximab in the conditioning regimen.

IV. To determine the effect of rituximab addition to the conditioning regimen on recovery of T regulatory (T-reg) cells, and to determine the effect of T-cell, including T-reg, number in the stem cell product and at day 30 on the incidence of grade II-IV acute GVHD (aGVHD) and the cumulative infectious complications at day 100.

V. To determine the effect of rituximab addition to the conditioning regimen on antigen presenting myeloid cell recovery, and to determine the effect of dendritic cell subset DC1, DC2 and myeloid-derived suppressor cells (MDSC), number in the stem cell graft and at day +30 on the incidence of acute GVHD grade II-IV and the cumulative incidence of infectious complications at day 100.

OUTLINE:

CONDITIONING REGIMEN: Patients receive one of the following conditioning regimens as per the transplant physician: cyclophosphamide and total-body irradiation (TBI); targeted busulfan and fludarabine; reduced-dose busulfan and fludarabine; or fludarabine and TBI.

GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS: Patients receive rituximab intravenously (IV) on days -6, 1, 8, and 15 and anti-thymocyte globulin IV over 6-8 hours on days -3 to -1. Patients also receive tacrolimus IV continuously and then orally (PO) beginning on day -1 and continuing until day 150 followed by a taper until day 180 and mycophenolate mofetil PO or IV twice daily on days -1 to 60.

TRANSPLANTATION: Patients undergo allogeneic hematopoietic stem cell transplantation on day 0.

Patients are followed up periodically for 100 days after transplant.

02

Conditions studied

  • Accelerated Phase Chronic Myelogenous Leukemia
  • Adult Acute Lymphoblastic Leukemia in Remission
  • Adult Acute Myeloid Leukemia in Remission
  • Adult Nasal Type Extranodal NK/T-cell Lymphoma
  • Blastic Phase Chronic Myelogenous Leukemia
  • Contiguous Stage II Adult Burkitt Lymphoma
  • Contiguous Stage II Adult Diffuse Large Cell Lymphoma
  • Contiguous Stage II Adult Diffuse Mixed Cell Lymphoma
  • Contiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma
  • Contiguous Stage II Adult Immunoblastic Large Cell Lymphoma
  • Contiguous Stage II Adult Lymphoblastic Lymphoma
  • Contiguous Stage II Grade 1 Follicular Lymphoma
  • Contiguous Stage II Grade 2 Follicular Lymphoma
  • Contiguous Stage II Grade 3 Follicular Lymphoma
  • Contiguous Stage II Mantle Cell Lymphoma
  • Contiguous Stage II Marginal Zone Lymphoma
  • Contiguous Stage II Small Lymphocytic Lymphoma
  • de Novo Myelodysplastic Syndromes
  • Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue
  • Graft Versus Host Disease
  • Nodal Marginal Zone B-cell Lymphoma
  • Noncontiguous Stage II Adult Burkitt Lymphoma
  • Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma
  • Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma
  • Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma
  • Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma
  • Noncontiguous Stage II Adult Lymphoblastic Lymphoma
  • Noncontiguous Stage II Grade 1 Follicular Lymphoma
  • Noncontiguous Stage II Grade 2 Follicular Lymphoma
  • Noncontiguous Stage II Grade 3 Follicular Lymphoma
  • Noncontiguous Stage II Mantle Cell Lymphoma
  • Noncontiguous Stage II Marginal Zone Lymphoma
  • Noncontiguous Stage II Small Lymphocytic Lymphoma
  • Previously Treated Myelodysplastic Syndromes
  • Recurrent Adult Acute Lymphoblastic Leukemia
  • Recurrent Adult Acute Myeloid Leukemia
  • Recurrent Adult Burkitt Lymphoma
  • Recurrent Adult Diffuse Large Cell Lymphoma
  • Recurrent Adult Diffuse Mixed Cell Lymphoma
  • Recurrent Adult Diffuse Small Cleaved Cell Lymphoma
  • Recurrent Adult Grade III Lymphomatoid Granulomatosis
  • Recurrent Adult Immunoblastic Large Cell Lymphoma
  • Recurrent Adult Lymphoblastic Lymphoma
  • Recurrent Adult T-cell Leukemia/Lymphoma
  • Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma
  • Recurrent Grade 1 Follicular Lymphoma
  • Recurrent Grade 2 Follicular Lymphoma
  • Recurrent Grade 3 Follicular Lymphoma
  • Recurrent Mantle Cell Lymphoma
  • Recurrent Marginal Zone Lymphoma
  • Recurrent Mycosis Fungoides/Sezary Syndrome
  • Recurrent Small Lymphocytic Lymphoma
  • Refractory Chronic Lymphocytic Leukemia
  • Relapsing Chronic Myelogenous Leukemia
  • Secondary Myelodysplastic Syndromes
  • Splenic Marginal Zone Lymphoma
  • Stage I Adult Burkitt Lymphoma
  • Stage I Adult Diffuse Large Cell Lymphoma
  • Stage I Adult Diffuse Mixed Cell Lymphoma
  • Stage I Adult Diffuse Small Cleaved Cell Lymphoma
  • Stage I Adult Immunoblastic Large Cell Lymphoma
  • Stage I Adult Lymphoblastic Lymphoma
  • Stage I Adult T-cell Leukemia/Lymphoma
  • Stage I Chronic Lymphocytic Leukemia
  • Stage I Cutaneous T-cell Non-Hodgkin Lymphoma
  • Stage I Grade 1 Follicular Lymphoma
  • Stage I Grade 2 Follicular Lymphoma
  • Stage I Grade 3 Follicular Lymphoma
  • Stage I Mantle Cell Lymphoma
  • Stage I Marginal Zone Lymphoma
  • Stage I Mycosis Fungoides/Sezary Syndrome
  • Stage I Small Lymphocytic Lymphoma
  • Stage II Adult T-cell Leukemia/Lymphoma
  • Stage II Chronic Lymphocytic Leukemia
  • Stage II Cutaneous T-cell Non-Hodgkin Lymphoma
  • Stage II Mycosis Fungoides/Sezary Syndrome
  • Stage III Adult Burkitt Lymphoma
  • Stage III Adult Diffuse Large Cell Lymphoma
  • Stage III Adult Diffuse Mixed Cell Lymphoma
  • Stage III Adult Diffuse Small Cleaved Cell Lymphoma
  • Stage III Adult Immunoblastic Large Cell Lymphoma
  • Stage III Adult Lymphoblastic Lymphoma
  • Stage III Adult T-cell Leukemia/Lymphoma
  • Stage III Chronic Lymphocytic Leukemia
  • Stage III Cutaneous T-cell Non-Hodgkin Lymphoma
  • Stage III Grade 1 Follicular Lymphoma
  • Stage III Grade 2 Follicular Lymphoma
  • Stage III Grade 3 Follicular Lymphoma
  • Stage III Mantle Cell Lymphoma
  • Stage III Marginal Zone Lymphoma
  • Stage III Mycosis Fungoides/Sezary Syndrome
  • Stage III Small Lymphocytic Lymphoma
  • Stage IV Adult Burkitt Lymphoma
  • Stage IV Adult Diffuse Large Cell Lymphoma
  • Stage IV Adult Diffuse Mixed Cell Lymphoma
  • Stage IV Adult Diffuse Small Cleaved Cell Lymphoma
  • Stage IV Adult Immunoblastic Large Cell Lymphoma
  • Stage IV Adult Lymphoblastic Lymphoma
  • Stage IV Adult T-cell Leukemia/Lymphoma
  • Stage IV Chronic Lymphocytic Leukemia
  • Stage IV Cutaneous T-cell Non-Hodgkin Lymphoma
  • Stage IV Grade 1 Follicular Lymphoma
  • Stage IV Grade 2 Follicular Lymphoma
  • Stage IV Grade 3 Follicular Lymphoma
  • Stage IV Mantle Cell Lymphoma
  • Stage IV Marginal Zone Lymphoma
  • Stage IV Mycosis Fungoides/Sezary Syndrome
  • Stage IV Small Lymphocytic Lymphoma
  • Untreated Adult Acute Lymphoblastic Leukemia
  • Untreated Adult Acute Myeloid Leukemia
  • Waldenström Macroglobulinemia
03

Who can participate

Ages eligible
19 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Diagnosis of acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myelogenous leukemia (CML) in accelerated phase or blast crisis, chronic lymphocytic leukemia (CLL), Non-Hodgkin lymphoma (NHL), myelodysplastic syndromes (MDS) (intermediate-2 or high-risk disease by International Pelvic Pain Society [IPPS])
  • Patients with AML, ALL, or MDS scheduled for myeloablative conditioning should have =\< 10% blasts in bone marrow or peripheral blood at the start of conditioning
  • Patients with AML, ALL, or CML scheduled for reduced intensity conditioning or non-myeloablative conditioning should be in complete morphologic remission at the start of conditioning (residual disease by flow cytometry or cytogenetics and/or incomplete recovery of neutrophil or platelet count are acceptable)
  • Patients with CLL or NHL scheduled for reduced intensity or non-myeloablative conditioning should have no evidence of bulky disease (> 50% bone marrow involvement or masses > 10 cm) at the start of conditioning
  • Fulfills all pulmonary, cardiac, renal, and hepatic criteria for standard-of-care matched unrelated donor (MUD) allogeneic HCT
  • Men and women of reproductive potential must agree to use an acceptable method of birth control during treatment and for twelve months after stem cell transplantation
  • Adequately matched unrelated donor available
  • Written informed consent; written informed consent of the unrelated donor is required to participate in the optional studies

Exclusion criteria

Exclusion Criteria:

  • Patient or donor infected with human immunodeficiency virus (HIV)
  • Patient or donor with history of hepatitis B or C and/or positive serology consistent with previous hepatitis B or C infection (patients and/or donor who received Hepatitis B vaccination are acceptable)
  • Patient or donor with active hepatitis B or C and/or detectable viral ribonucleic acid (RNA)
  • More than 20,000 circulating CD20+ cells/uL
  • Treatment with rituximab for any reason in the 12 months preceding HCT
  • Patient scheduled for cord blood transplantation
  • Presence of active uncontrolled infection at start of conditioning
  • Presence of active central nervous system (CNS) disease (history of adequately treated CNS disease is acceptable)
  • Presence of uncontrolled psychiatric disorder
  • Patient unable to give informed consent
  • Unrelated donor products received from the Deutsche Knochenmarkspenderdatei (DKMS) Registry are not eligible for the optional study
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
20 participants (actual)

Study arms

  • Experimental
    Treatment (Rituximab and allogeneic HCT transplant)

    CONDITIONING REGIMEN: Patients receive one of the following conditioning regimens as per the transplant physician: cyclophosphamide and TBI; targeted busulfan and fludarabine; reduced-dose busulfan and fludarabine; or fludarabine and TBI. GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS: Patients receive rituximab IV on days -6, 1, 8, and 15 and anti-thymocyte globulin IV over 6-8 hours on days -3 to -1. Patients also receive tacrolimus IV continuously and then PO beginning on day -1 and continuing until day 150 followed by a taper until day 180 and mycophenolate mofetil PO or IV twice daily on days -1 to 60. TRANSPLANTATION: Patients undergo allogeneic hematopoietic stem cell transplantation on day 0.

    Drug: rituximab · Drug: mycophenolate mofetil · Drug: tacrolimus · Drug: anti-thymocyte globulin · Procedure: allogeneic hematopoietic stem cell transplantation · Other: laboratory biomarker analysis · Biological: graft versus host disease prophylaxis/therapy · Drug: cyclophosphamide · Drug: fludarabine phosphate · Drug: busulfan · Radiation: total-body irradiation · Biological: graft-versus-tumor induction therapy · Biological: immunosuppressive therapy

Interventions

  • Drugrituximab

    Given IV

    Also known as: IDEC-C2B8, IDEC-C2B8 monoclonal antibody, Mabthera, MOAB IDEC-C2B8, Rituxan

  • Drugmycophenolate mofetil

    Given IV or PO

    Also known as: Cellcept, MMF

  • Drugtacrolimus

    Given IV

    Also known as: FK 506, Prograf

  • Druganti-thymocyte globulin

    Given IV

    Also known as: ATG, ATGAM, lymphocyte immune globulin, Thymoglobulin

  • Procedureallogeneic hematopoietic stem cell transplantation

    Stem cell transplant

  • Otherlaboratory biomarker analysis

    Correlative studies

  • Biologicalgraft versus host disease prophylaxis/therapy

    Undergo graft versus host disease prophylaxis/therapy

    Also known as: prophylaxis/therapy, graft versus host disease, prophylaxis/therapy, GVHD

  • Drugcyclophosphamide

    Given PO or IV

    Also known as: CPM, CTX, Cytoxan, Endoxan, Endoxana

  • Drugfludarabine phosphate

    Given IV

    Also known as: 2-F-ara-AMP, Beneflur, Fludara

  • Drugbusulfan

    Given IV

    Also known as: BSF, BU, Misulfan, Mitosan, Myeloleukon

  • Radiationtotal-body irradiation

    Undergo TBI

    Also known as: TBI

  • Biologicalgraft-versus-tumor induction therapy

    Undergo graft-versus-tumor induction therapy

    Also known as: graft versus host disease induction, graft versus tumor induction

  • Biologicalimmunosuppressive therapy

    Undergo immunosuppressive therapy

    Also known as: immunosuppression

05

What researchers measure

Primary outcomes

  1. Number of Participants With Grades II-IV Acute GVHD

    Determined with death as a competing risk. Defined and staged using the 1994 consensus conference modifications of the Glucksberg criteria.

    Time frame: At day 100

Secondary outcomes

  1. Event-free Survival

    Estimated using Kaplan-Meier estimator.

    Time frame: From the date of transplant with relapse/progression or death as censored events, up to 2 years

  2. Overall Survival

    Estimated using Kaplan-Meier estimator.

    Time frame: From the date of transplant with death from any cause as a censored event, up to 2 years

  3. Transplant-related Mortality (TRM)

    Transplant-related mortality (TRM) defined as any mortality after transplantation except mortality from relapsed disease - Reported as a simple percentage.

    Time frame: At day 100

06

Results

Posted Feb 26, 2019

Participant flow

Participant flow — Overall Study
MilestoneTreatment (Rituximab and Allogeneic HCT Transplant)
Started20
Completed18
Not completed2
Withdrew: Ineiligible2

Outcome measures

PrimaryNumber of Participants With Grades II-IV Acute GVHD

Determined with death as a competing risk. Defined and staged using the 1994 consensus conference modifications of the Glucksberg criteria.

Time frame:
At day 100
Reported as:
Count of participants · Participants
Number of Participants With Grades II-IV Acute GVHD
ParticipantsTreatment (Rituximab and Allogeneic HCT Transplant)
Number of Participants With Grades II-IV Acute GVHD16
SecondaryEvent-free Survival

Estimated using Kaplan-Meier estimator.

Time frame:
From the date of transplant with relapse/progression or death as censored events, up to 2 years
Reported as:
Median · months
Event-free Survival
monthsTreatment (Rituximab and Allogeneic HCT Transplant)
Event-free Survival12 (6 to 18)
SecondaryOverall Survival

Estimated using Kaplan-Meier estimator.

Time frame:
From the date of transplant with death from any cause as a censored event, up to 2 years
Reported as:
Median · months
Overall Survival
monthsTreatment (Rituximab and Allogeneic HCT Transplant)
Overall Survival12 (9 to 18)
SecondaryTransplant-related Mortality (TRM)

Transplant-related mortality (TRM) defined as any mortality after transplantation except mortality from relapsed disease - Reported as a simple percentage.

Time frame:
At day 100
Reported as:
Count of participants · Participants
Transplant-related Mortality (TRM)
ParticipantsTreatment (Rituximab and Allogeneic HCT Transplant)
Transplant-related Mortality (TRM)0

Adverse events

Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Treatment (Rituximab and Allogeneic HCT Transplant)5/20 (25%)10/20 (50%)16/20 (80%)
Most frequent serious events
Showing 10 of 14
Most frequent serious events
EventTreatment (Rituximab and Allogeneic HCT Transplant)
DiarrheaGastrointestinal disorders3/20
pneumonitisInfections and infestations3/20
sepsisInfections and infestations3/20
acute kidney injuryRenal and urinary disorders3/20
cystitisInfections and infestations3/20
Immune system disorders-otherImmune system disorders2/20
vomitingGastrointestinal disorders1/20
anorexiaGastrointestinal disorders1/20
mucositis oralGastrointestinal disorders1/20
ileusGastrointestinal disorders1/20
Most frequent other events
Showing 10 of 31
Most frequent other events
EventTreatment (Rituximab and Allogeneic HCT Transplant)
HyperglycemiaMetabolism and nutrition disorders8/20
HypokalemiaMetabolism and nutrition disorders7/20
HyponatremiaMetabolism and nutrition disorders5/20
SepsisInfections and infestations5/20
HypophosphatemiaMetabolism and nutrition disorders4/20
HypoalbuminemiaMetabolism and nutrition disorders4/20
Febrile NeutropeniaBlood and lymphatic system disorders3/20
AnorexiaMetabolism and nutrition disorders3/20
Mucositis - oralGastrointestinal disorders3/20
RashInfections and infestations2/20

Baseline characteristics

Age, Continuous
Age, Continuous(years)Treatment (Rituximab and Allogeneic HCT Transplant)
Median39 (21 to 52)
Sex: Female, Male
Sex: Female, Male(Participants)Treatment (Rituximab and Allogeneic HCT Transplant)
Female11
Male9
Region of Enrollment
Region of Enrollment(participants)Treatment (Rituximab and Allogeneic HCT Transplant)
United States20
07

Study locations

1 site
  • University of Nebraska Medical Center
    Omaha, Nebraska 68198, United States
08

Registry details

Key details

Study ID
NCT01044745
Lead sponsor
University of Nebraska
Collaborators
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Jan 8, 2010
Start date
Dec 10, 2009
Primary completion
Dec 19, 2012
Completion
Oct 10, 2017
Results posted
Feb 26, 2019
Last update
Sep 6, 2023

Study contacts

Robert Bociek, MD
principal investigator · University of Nebraska

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is terminated, as verified in Aug 2023. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Start the discussion