Tacrolimus and Mycophenolate Mofetil in Preventing Acute Graft-Versus-Host Disease in Patients With Advanced Hematologic Cancer Who Are Undergoing Donor Stem Cell Transplantation

This study has been completed.
National Cancer Institute (NCI)
Information provided by:
Fred Hutchinson Cancer Research Center
ClinicalTrials.gov Identifier:
First received: November 9, 2004
Last updated: May 12, 2010
Last verified: May 2010

RATIONALE: Tacrolimus and mycophenolate mofetil may be an effective treatment for graft-versus-host disease caused by donor stem cell transplantation.

PURPOSE: This phase II trial is studying how well giving tacrolimus together with mycophenolate mofetil works in preventing acute graft-versus-host disease in patients who are undergoing donor stem cell transplantation for advanced hematologic cancer.

Condition Intervention Phase
Chronic Myeloproliferative Disorders
Graft Versus Host Disease
Multiple Myeloma and Plasma Cell Neoplasm
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Diseases
Drug: mycophenolate mofetil
Drug: tacrolimus
Phase 2

Study Type: Interventional
Study Design: Masking: Open Label
Primary Purpose: Supportive Care
Official Title: A Phase II Study of the Combination of Tacrolimus and Mycophenolate Mofetil for the Prevention of Acute Graft-Versus-Host Disease

Resource links provided by NLM:

Genetic and Rare Diseases Information Center resources: Acute Graft Versus Host Disease Acute Lymphoblastic Leukemia Acute Myeloid Leukemia Acute Non Lymphoblastic Leukemia Anaplastic Plasmacytoma B-cell Lymphomas Burkitt Lymphoma Childhood Acute Lymphoblastic Leukemia Chronic Lymphocytic Leukemia Chronic Myeloid Leukemia Chronic Myelomonocytic Leukemia Chronic Myeloproliferative Disorders Chronic Neutrophilic Leukemia Cutaneous T-cell Lymphoma Follicular Lymphoma Hairy Cell Leukemia Hodgkin Lymphoma Hodgkin Lymphoma, Childhood Homologous Wasting Disease Juvenile Myelomonocytic Leukemia Leukemia, B-cell, Chronic Lymphoblastic Lymphoma Lymphoma, Large-cell Lymphoma, Large-cell, Immunoblastic Lymphoma, Small Cleaved-cell, Diffuse Lymphosarcoma Mantle Cell Lymphoma Multiple Myeloma Mycosis Fungoides Myelodysplastic Syndromes Myelodysplastic/myeloproliferative Disease Myelofibrosis Myeloid Leukemia Plasmablastic Lymphoma Sezary Syndrome Sideroblastic Anemia Pyridoxine-refractory Autosomal Recessive Small Non-cleaved Cell Lymphoma Squamous Cell Carcinoma of the Head and Neck
U.S. FDA Resources

Further study details as provided by Fred Hutchinson Cancer Research Center:

Primary Outcome Measures:
  • Efficacy

Secondary Outcome Measures:
  • Safety
  • Area under the curve of plasma mycophenolic acid

Study Start Date: August 2004
Primary Completion Date: May 2006 (Final data collection date for primary outcome measure)
Detailed Description:



  • Determine the efficacy of tacrolimus and mycophenolate mofetil in preventing acute graft-vs-host disease in patients with advanced hematologic malignancies undergoing allogeneic peripheral blood stem cell transplantation.


  • Determine the safety of this regimen in these patients.

OUTLINE: This is a non-randomized study.

Patients receive tacrolimus IV continuously or orally twice daily on days -1 to 60 followed by a taper to day 180. Patients also receive mycophenolate mofetil IV over 2 hours or orally twice daily on days 0-60 followed by a taper to day 180. Treatment is discontinued at day 180 in the absence of graft-vs-host disease.

Patients are followed for survival.

PROJECTED ACCRUAL: A total of 5-25 patients will be accrued for this study.


Ages Eligible for Study:   up to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • Diagnosis of 1 of the following hematologic malignancies:

    • Acute myeloid leukemia beyond first complete remission (CR1)
    • Acute lymphoblastic leukemia beyond CR1
    • Chronic myelogenous leukemia in second chronic phase, accelerated phase, or blastic phase
    • Non-Hodgkin's lymphoma beyond CR2
    • Hodgkin's lymphoma beyond CR2
    • Multiple myeloma (any stage)
    • Myelodysplastic syndromes beyond refractory anemia (including chronic myelomonocytic leukemia)
    • Any refractory hematologic malignancy
  • Advanced disease
  • Scheduled for transplantation of mobilized allogeneic peripheral blood stem cells

    • Genotypically HLA-identical stem cell donor available



  • 65 and under

Performance status

  • Not specified

Life expectancy

  • Not specified


  • Not specified


  • Bilirubin ≤ 1.5 times upper limit of normal (ULN)
  • SGOT and SGPT ≤ 2.0 times ULN


  • Creatinine clearance ≥ 60 mL/min


  • No acute pulmonary infection by chest x-ray
  • No severe hypoxemia (partial O_2 < 70% of predicted) AND DLCO < 70% of predicted
  • No mild hypoxemia (partial O_2 < 80% of predicted) AND DLCO < 60% of predicted


  • Not pregnant or nursing
  • Negative pregnancy test
  • No active systemic infection not controlled with antimicrobial therapy
  • HIV negative (HIV-1 or other virus)


Biologic therapy

  • No concurrent T-cell depleted hematopoietic stem cell graft


  • Not specified

Endocrine therapy

  • Not specified


  • Not specified


  • Not specified
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00096096

United States, Washington
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98109-1024
Sponsors and Collaborators
Fred Hutchinson Cancer Research Center
National Cancer Institute (NCI)
Principal Investigator: Richard Nash, MD Fred Hutchinson Cancer Research Center
  More Information

Additional Information:
No publications provided

ClinicalTrials.gov Identifier: NCT00096096     History of Changes
Other Study ID Numbers: 1893.00, FHCRC-1893.00, CDR0000391026
Study First Received: November 9, 2004
Last Updated: May 12, 2010
Health Authority: United States: Federal Government
United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by Fred Hutchinson Cancer Research Center:
graft versus host disease
accelerated phase chronic myelogenous leukemia
relapsing chronic myelogenous leukemia
recurrent adult Hodgkin lymphoma
refractory multiple myeloma
recurrent adult Burkitt lymphoma
chronic idiopathic myelofibrosis
recurrent adult diffuse large cell lymphoma
recurrent adult diffuse mixed cell lymphoma
recurrent adult diffuse small cleaved cell lymphoma
recurrent adult immunoblastic large cell lymphoma
recurrent mantle cell lymphoma
recurrent grade 1 follicular lymphoma
recurrent grade 2 follicular lymphoma
recurrent grade 3 follicular lymphoma
recurrent adult lymphoblastic lymphoma
secondary acute myeloid leukemia
secondary myelodysplastic syndromes
adult acute lymphoblastic leukemia in remission
de novo myelodysplastic syndromes
previously treated myelodysplastic syndromes
chronic myelomonocytic leukemia
refractory anemia with excess blasts in transformation
refractory anemia with excess blasts
refractory anemia with ringed sideroblasts
refractory hairy cell leukemia
juvenile myelomonocytic leukemia
chronic eosinophilic leukemia
chronic neutrophilic leukemia
chronic phase chronic myelogenous leukemia

Additional relevant MeSH terms:
Graft vs Host Disease
Multiple Myeloma
Myelodysplastic Syndromes
Myelodysplastic-Myeloproliferative Diseases
Myeloproliferative Disorders
Neoplasms, Plasma Cell
Blood Protein Disorders
Bone Marrow Diseases
Cardiovascular Diseases
Hematologic Diseases
Hemorrhagic Disorders
Hemostatic Disorders
Immune System Diseases
Immunoproliferative Disorders
Lymphoproliferative Disorders
Neoplasms by Histologic Type
Precancerous Conditions
Vascular Diseases
Mycophenolate mofetil
Mycophenolic Acid
Antibiotics, Antineoplastic
Antineoplastic Agents
Enzyme Inhibitors
Immunologic Factors
Immunosuppressive Agents

ClinicalTrials.gov processed this record on October 09, 2015