NCT00089141

Brief Summary

RATIONALE: Mycophenolate mofetil added to immunosuppressive treatment regimens may be effective in treating newly diagnosed chronic graft-versus-host disease caused by stem cell transplantation. It is not yet known whether immunosuppressive treatment regimens are more effective with or without mycophenolate mofetil in treating chronic graft-versus-host disease. PURPOSE: This randomized phase III trial is studying whether the addition of mycophenolate mofetil improves the efficacy of immunosuppressive treatment regimens in patients with newly diagnosed chronic graft-versus-host disease.

Trial Health

60
Monitor

Trial Health Score

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

Enrollment
151

participants targeted

Target at P25-P50 for phase_3 cancer

Timeline
Completed

Started May 2004

Typical duration for phase_3 cancer

Geographic Reach
2 countries

16 active sites

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

Study Start

First participant enrolled

May 1, 2004

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

August 4, 2004

Completed
1 day until next milestone

First Posted

Study publicly available on registry

August 5, 2004

Completed
3.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2008

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2008

Completed
12 months until next milestone

Results Posted

Study results publicly available

August 26, 2009

Completed
Last Updated

May 3, 2013

Status Verified

August 1, 2009

Enrollment Period

4.2 years

First QC Date

August 4, 2004

Results QC Date

July 15, 2009

Last Update Submit

May 1, 2013

Conditions

Cancer

Keywords

graft versus host diseaseaccelerated phase chronic myelogenous leukemiaadult acute lymphoblastic leukemia in remissionadult acute myeloid leukemia in remissionchildhood acute lymphoblastic leukemia in remissionchildhood acute myeloid leukemia in remissionatypical chronic myeloid leukemiablastic phase chronic myelogenous leukemiachildhood chronic myelogenous leukemiachronic eosinophilic leukemiachronic idiopathic myelofibrosischronic myelomonocytic leukemiachronic neutrophilic leukemiachronic phase chronic myelogenous leukemiade novo myelodysplastic syndromesdisseminated neuroblastomaextranodal marginal zone B-cell lymphoma of mucosa-associated lymphoid tissuejuvenile myelomonocytic leukemiamyelodysplastic/myeloproliferative disease, unclassifiablenodal marginal zone B-cell lymphomanoncontiguous stage II adult Burkitt lymphomanoncontiguous stage II adult diffuse large cell lymphomanoncontiguous stage II adult diffuse mixed cell lymphomanoncontiguous stage II adult diffuse small cleaved cell lymphomanoncontiguous stage II adult immunoblastic large cell lymphomanoncontiguous stage II adult lymphoblastic lymphomanoncontiguous stage II grade 1 follicular lymphomanoncontiguous stage II grade 2 follicular lymphomanoncontiguous stage II grade 3 follicular lymphomanoncontiguous stage II mantle cell lymphomanoncontiguous stage II marginal zone lymphomanoncontiguous stage II small lymphocytic lymphomapoor prognosis metastatic gestational trophoblastic tumorpreviously treated childhood rhabdomyosarcomapreviously treated myelodysplastic syndromessecondary acute myeloid leukemiasecondary myelodysplastic syndromessplenic marginal zone lymphomastage I multiple myelomastage II multiple myelomastage II ovarian epithelial cancerstage III adult Burkitt lymphomastage III adult diffuse large cell lymphomastage III adult diffuse mixed cell lymphomastage III adult diffuse small cleaved cell lymphomastage III adult immunoblastic large cell lymphomastage III adult lymphoblastic lymphomastage III chronic lymphocytic leukemiastage III grade 1 follicular lymphomastage III grade 2 follicular lymphomastage III grade 3 follicular lymphomastage III mantle cell lymphomastage III marginal zone lymphomastage III multiple myelomastage III ovarian epithelial cancerstage III small lymphocytic lymphomastage III malignant testicular germ cell tumorstage IIIA breast cancerstage IIIB breast cancerstage IIIC breast cancerstage IV adult Burkitt lymphomastage IV adult diffuse large cell lymphomastage IV adult diffuse mixed cell lymphomastage IV adult diffuse small cleaved cell lymphomastage IV adult immunoblastic large cell lymphomastage IV adult lymphoblastic lymphomastage IV breast cancerstage IV chronic lymphocytic leukemiastage IV grade 1 follicular lymphomastage IV grade 2 follicular lymphomastage IV grade 3 follicular lymphomastage IV mantle cell lymphomastage IV marginal zone lymphomastage IV ovarian epithelial cancerstage IV small lymphocytic lymphomachildhood myelodysplastic syndromes

Outcome Measures

Primary Outcomes (1)

  • Cure of Chronic GVHD Without Resorting to Secondary Systemic Therapy

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

    2 years

Secondary Outcomes (9)

  • Definitive Absence of Efficacy Success

    2 years

  • Open Label Systemic Treatment Because of Inadequate Response to Primary Therapy

    2 years

  • Bronchiolitis Obliterans

    within 4 years

  • Recurrent Malignancy

    within 4 years

  • Non-relapse Mortality

    within 4 years

  • +4 more secondary outcomes

Study Arms (2)

Mycophenolate mofetil

ACTIVE COMPARATOR

Patients receive oral mycophenolate mofetil twice daily.

Drug: mycophenolate mofetil

Placebo

PLACEBO COMPARATOR

Patients receive oral placebo twice daily

Drug: placebo

Interventions

mycophenolate mofetilDRUG

Given orally

Also known as: CellCept
Mycophenolate mofetil
placeboDRUG

Given orally

Also known as: Control
Placebo

Eligibility Criteria

Age4 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (16)

City of Hope Comprehensive Cancer Center

Duarte, California, 91010-3000, United States

Location

Stanford Cancer Center

Stanford, California, 94305-5824, United States

Location

University of Florida Shands Cancer Center

Gainesville, Florida, 32610-100277, United States

Location

University of Chicago Cancer Research Center

Chicago, Illinois, 60637-1470, United States

Location

University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, 48109-0942, United States

Location

Masonic Cancer Center at University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

UNMC Eppley Cancer Center at the University of Nebraska Medical Center

Omaha, Nebraska, 68198-3330, United States

Location

Hackensack University Medical Center Cancer Center

Hackensack, New Jersey, 07601, United States

Location

Oregon Health and Science University Cancer Institute

Portland, Oregon, 97239-3098, United States

Location

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, 37232-6838, United States

Location

Baylor University Medical Center - Dallas

Dallas, Texas, 75246, United States

Location

M. D. Anderson Cancer Center at University of Texas

Houston, Texas, 77030-4009, United States

Location

Texas Transplant Institute

San Antonio, Texas, 78229, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109-1024, United States

Location

University of Washington School of Medicine

Seattle, Washington, 98195, United States

Location

Princess Margaret Hospital

Toronto, Ontario, M5G 2M9, Canada

Location

Related Publications (1)

  • Martin PJ, Storer BE, Rowley SD, Flowers ME, Lee SJ, Carpenter PA, Wingard JR, Shaughnessy PJ, DeVetten MP, Jagasia M, Fay JW, van Besien K, Gupta V, Kitko C, Johnston LJ, Maziarz RT, Arora M, Jacobson PA, Weisdorf D. Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease. Blood. 2009 May 21;113(21):5074-82. doi: 10.1182/blood-2009-02-202937. Epub 2009 Mar 6.

    PMID: 19270260RESULT

MeSH Terms

Conditions

NeoplasmsGraft vs Host DiseaseLeukemia, Myeloid, Accelerated PhaseLeukemia, Myeloid, Chronic, Atypical, BCR-ABL NegativeBlast CrisisPdgfra-Associated Chronic Eosinophilic LeukemiaPrimary MyelofibrosisLeukemia, Myelomonocytic, ChronicLeukemia, Neutrophilic, ChronicLeukemia, Myeloid, Chronic-PhaseLeukemia, Myelomonocytic, JuvenileMyeloproliferative DisordersLymphoma, B-Cell, Marginal ZoneMultiple MyelomaCarcinoma, Ovarian EpithelialBurkitt LymphomaLymphoma, Large B-Cell, DiffuseLymphoma, Non-HodgkinLymphoma, Large-Cell, ImmunoblasticPrecursor Cell Lymphoblastic Leukemia-LymphomaLeukemia, Lymphocytic, Chronic, B-CellLymphoma, FollicularLymphoma, Mantle-CellTesticular NeoplasmsBreast Neoplasms

Interventions

Mycophenolic Acid

Condition Hierarchy (Ancestors)

Immune System DiseasesLeukemia, Myelogenous, Chronic, BCR-ABL PositiveLeukemia, MyeloidLeukemiaNeoplasms by Histologic TypeBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsMyelodysplastic-Myeloproliferative DiseasesCell Transformation, NeoplasticCarcinogenesisNeoplastic ProcessesLymphoma, B-CellLymphomaLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersNeoplasms, Plasma CellHemostatic DisordersVascular DiseasesCardiovascular DiseasesParaproteinemiasBlood Protein DisordersHemorrhagic DisordersCarcinomaNeoplasms, Glandular and EpithelialOvarian NeoplasmsEndocrine Gland NeoplasmsNeoplasms by SiteOvarian DiseasesAdnexal DiseasesGenital Diseases, FemaleFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesGenital Neoplasms, FemaleUrogenital NeoplasmsGenital DiseasesEndocrine System DiseasesGonadal DisordersEpstein-Barr Virus InfectionsHerpesviridae InfectionsDNA Virus InfectionsVirus DiseasesInfectionsTumor Virus InfectionsLeukemia, LymphoidLeukemia, B-CellGenital Neoplasms, MaleGenital Diseases, MaleMale Urogenital DiseasesTesticular DiseasesBreast DiseasesSkin DiseasesSkin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

CaproatesAcids, AcyclicCarboxylic AcidsOrganic ChemicalsFatty AcidsLipids

Results Point of Contact

Title
Paul J. Martin, M.D.
Organization
Fred Hutchinson Cancer Research Center (FHCRC)
pmartin@fhcrc.org
206-667-4798

Study Officials

  • Paul J. Martin, MD

    Fred Hutchinson Cancer Center

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Member

Study Record Dates

First Submitted

August 4, 2004

First Posted

August 5, 2004

Study Start

May 1, 2004

Primary Completion

July 1, 2008

Study Completion

September 1, 2008

Last Updated

May 3, 2013

Results First Posted

August 26, 2009

Record last verified: 2009-08

Locations

US(15)CA(1)