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A Randomized Study to Evaluate The Efficacy of Mycophenolate Mofetil Added to The Systemic Immunosuppressive Regimen First Used For Treatment of Chronic Graft-Versus-Host Disease


Phase 3
4 Years
N/A
Not Enrolling
Both
Cancer

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Trial Information

A Randomized Study to Evaluate The Efficacy of Mycophenolate Mofetil Added to The Systemic Immunosuppressive Regimen First Used For Treatment of Chronic Graft-Versus-Host Disease


OBJECTIVES:

- Compare the efficacy of immunosuppressive treatment regimens with vs without
mycophenolate mofetil in patients with newly diagnosed chronic graft-vs-host disease.

- Compare the quality of life of patients treated with these regimens.

OUTLINE: This is a randomized, double-blind, placebo-controlled, prospective, multicenter
study. Patients are stratified according to organ involvement of chronic graft-versus-host
disease (GVHD) (single organ vs multiple organs) and transplant center. Patients are
randomized to 1 of 2 treatment arms.

All patients receive usual therapy for chronic GVHD comprising oral prednisone twice daily
and oral cyclosporine, oral tacrolimus or oral sirolimus twice daily until 2 weeks after the
first evidence of improvement of symptoms of chronic GVHD.

- Arm I: Patients receive oral mycophenolate mofetil twice daily.

- Arm II: Patients receive oral placebo twice daily. In both arms administration of the
study drug continues for 3 months after completion of prednisone and cyclosporine,
tacrolimus or sirolimus in the absence of disease progression or unacceptable toxicity.

Quality of life is assessed at baseline and then every 3 months.

Patients are followed every 3 months for 3-5 years.

PROJECTED ACCRUAL: A total of 230 patients (115 per treatment arm) will be accrued for this
study within 3 years.

Inclusion Criteria


DISEASE CHARACTERISTICS:

- Newly diagnosed chronic-graft-versus host disease (GVHD)

- Systemic immunosuppressive treatment indicated AND no contraindication to treatment
with mycophenolate mofetil

- Has undergone prior transplantation with any type of donor, hematopoietic stem cell
graft, or conditioning regimen

- No clinical, laboratory, or image-based evidence known to be present at the time of
enrollment and indicating a high probability of subsequent recurrent or progressive
disease

PATIENT CHARACTERISTICS:

Age

- Any age

Performance status

- Not specified

Life expectancy

- Not specified

Hematopoietic

- Absolute neutrophil count ≥ 1,500/mm^3

Hepatic

- Not specified

Renal

- Not specified

Pulmonary

- No known bronchiolitis obliterans as a manifestation of chronic GVHD

Immunologic

- No fungal infection without radiographic evidence of improvement during continued
antifungal therapy

- No cytomegalovirus (CMV) pneumonia without major radiographic evidence of improvement

- No other CMV infection without reduction of antigenemia or viral load during
continued antiviral therapy

- No active disseminated varicella zoster viral infection

- No known hypersensitivity or allergy to MMF

Gastrointestinal

- Able to tolerate oral medication

- No lactose-intolerant children who are too young to swallow capsules

- No frank blood from the rectum

- No melena

- No known gastrointestinal ulceration

Other

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception

- Female patients must use 2 forms of contraception 4 weeks prior to, during, and
for 6 weeks after completion of study treatment

- Not hospitalized at time of enrollment

- No rare, hereditary deficiency of hypoxanthine-guanine phosphoribosyl-transferase
(HGPRT)

PRIOR CONCURRENT THERAPY:

Biologic therapy

- See Disease Characteristics

Chemotherapy

- Not specified

Endocrine therapy

- Prior treatment with prednisone or equivalent allowed provided the dose was ≤ 1.0
mg/kg/day at the time of enrollment

- Concurrent systemic glucocorticoids allowed

Radiotherapy

- Not specified

Surgery

- Not specified

Other

- Prior mycophenolate mofetil (MMF) for prevention or treatment of acute GVHD allowed
provided MMF was discontinued at least 2 weeks before the diagnosis of chronic GVHD
was made

- No prior systemic treatment for chronic GVHD

- No prior treatment for chronic GVHD

- Concurrent antacids allowed provided there is at least a 2-hour interval before and
after administration of MMF

- No other concurrent systemic immunosuppressive treatment except cyclosporine,
tacrolimus or sirolimus

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Outcome Measure:

Cure of Chronic GVHD Without Resorting to Secondary Systemic Therapy

Outcome Description:

Withdrawal of all systemic immunosuppressive treatment after resolution of chronic GVHD, before death or onset of recurrent malignancy

Outcome Time Frame:

2 years

Safety Issue:

No

Principal Investigator

Paul J. Martin, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Fred Hutchinson Cancer Research Center

Authority:

United States: Food and Drug Administration

Study ID:

1697.00

NCT ID:

NCT00089141

Start Date:

May 2004

Completion Date:

September 2008

Related Keywords:

  • Cancer
  • graft versus host disease
  • accelerated phase chronic myelogenous leukemia
  • adult acute lymphoblastic leukemia in remission
  • adult acute myeloid leukemia in remission
  • childhood acute lymphoblastic leukemia in remission
  • childhood acute myeloid leukemia in remission
  • atypical chronic myeloid leukemia
  • blastic phase chronic myelogenous leukemia
  • childhood chronic myelogenous leukemia
  • chronic eosinophilic leukemia
  • chronic idiopathic myelofibrosis
  • chronic myelomonocytic leukemia
  • chronic neutrophilic leukemia
  • chronic phase chronic myelogenous leukemia
  • de novo myelodysplastic syndromes
  • disseminated neuroblastoma
  • extranodal marginal zone B-cell lymphoma of mucosa-associated lymphoid tissue
  • juvenile myelomonocytic leukemia
  • myelodysplastic/myeloproliferative disease, unclassifiable
  • 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
  • poor prognosis metastatic gestational trophoblastic tumor
  • previously treated childhood rhabdomyosarcoma
  • previously treated myelodysplastic syndromes
  • secondary acute myeloid leukemia
  • secondary myelodysplastic syndromes
  • splenic marginal zone lymphoma
  • stage I multiple myeloma
  • stage II multiple myeloma
  • stage II ovarian epithelial cancer
  • 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 chronic lymphocytic leukemia
  • 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 multiple myeloma
  • stage III ovarian epithelial cancer
  • stage III small lymphocytic lymphoma
  • stage III malignant testicular germ cell tumor
  • stage IIIA breast cancer
  • stage IIIB breast cancer
  • stage IIIC breast cancer
  • 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 breast cancer
  • stage IV chronic lymphocytic leukemia
  • 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 ovarian epithelial cancer
  • stage IV small lymphocytic lymphoma
  • childhood myelodysplastic syndromes
  • Graft vs Host Disease
  • Lymphoma, Non-Hodgkin
  • Lymphoma, Large-Cell, Immunoblastic

Name

Location

Fred Hutchinson Cancer Research CenterSeattle, Washington  98109
University of Michigan Comprehensive Cancer CenterAnn Arbor, Michigan  48109-0752
University of Chicago Cancer Research CenterChicago, Illinois  60637
Vanderbilt-Ingram Cancer CenterNashville, Tennessee  37232-6838
City of Hope Comprehensive Cancer CenterDuarte, California  91010
University of Florida Shands Cancer CenterGainesville, Florida  32610-0232
Hackensack University Medical Center Cancer CenterHackensack, New Jersey  07601
UNMC Eppley Cancer Center at the University of Nebraska Medical CenterOmaha, Nebraska  68198-7680
Masonic Cancer Center at University of MinnesotaMinneapolis, Minnesota  55455
M. D. Anderson Cancer Center at University of TexasHouston, Texas  77030-4009
University of Washington School of MedicineSeattle, Washington  98195
Stanford Cancer CenterStanford, California  94305-5824
Texas Transplant InstituteSan Antonio, Texas  78229
Oregon Health and Science University Cancer InstitutePortland, Oregon  97239-3098
Baylor University Medical Center - DallasDallas, Texas  75246