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CancerPhase 3NCT00089141

Mycophenolate Mofetil (MMF) for Treatment of Chronic Graft-versus-host Disease (GVHD)

A Phase 3 study testing mycophenolate mofetil in people with Cancer, run by Martin, Paul at 16 sites in 2 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 4 years and older
Conditions the study is for
Cancer
What is being tested?
mycophenolate mofetil
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
151 participants
Where
16 sitesin 2 countries
Study dates
May 2004to Sep 2008
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 4 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

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

Trial locations and contacts

16 sites in 2 countries.

16 sites in 2 countries

United States15 sites

  • University of Michigan Comprehensive Cancer Center

    Ann Arbor, Michigan, 48109-0942

  • University of Chicago Cancer Research Center

    Chicago, Illinois, 60637-1470

  • Baylor University Medical Center - Dallas

    Dallas, Texas, 75246

  • City of Hope Comprehensive Cancer Center

    Duarte, California, 91010-3000

  • University of Florida Shands Cancer Center

    Gainesville, Florida, 32610-100277

  • Hackensack University Medical Center Cancer Center

    Hackensack, New Jersey, 07601

  • M. D. Anderson Cancer Center at University of Texas

    Houston, Texas, 77030-4009

  • Masonic Cancer Center at University of Minnesota

    Minneapolis, Minnesota, 55455

  • Vanderbilt-Ingram Cancer Center

    Nashville, Tennessee, 37232-6838

  • UNMC Eppley Cancer Center at the University of Nebraska Medical Center

    Omaha, Nebraska, 68198-3330

  • Oregon Health and Science University Cancer Institute

    Portland, Oregon, 97239-3098

  • Texas Transplant Institute

    San Antonio, Texas, 78229

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington, 98109-1024

  • University of Washington School of Medicine

    Seattle, Washington, 98195

  • Stanford Cancer Center

    Stanford, California, 94305-5824

Other trials in United States

Canada1 site

  • Princess Margaret Hospital

    Toronto, Ontario, M5G 2M9

Other trials in Canada

About this study

Official title: 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

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.

Read the detailed description

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.

Conditions
Cancer
Being tested
mycophenolate mofetil, placebo
Keywords
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
Medical subject headings
Neoplasms, Graft vs Host Disease, Leukemia, Myeloid, Accelerated Phase, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Blast Crisis, Pdgfra-Associated Chronic Eosinophilic Leukemia, Primary Myelofibrosis, Leukemia, Myelomonocytic, Chronic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT00089141
Study groupRoleWhat they receive
Mycophenolate mofetilActive comparatorPatients receive oral mycophenolate mofetil twice daily.
Receives: Drug: mycophenolate mofetil
PlaceboPlacebo comparatorPatients receive oral placebo twice daily
Receives: Drug: placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: 2 years

Secondary outcomes (9)

  1. Definitive Absence of Efficacy Success

    Administration of secondary systemic therapy for chronic GVHD, death during primary therapy, or onset of recurrent malignancy or bronchiolitis obliterans during primary therapy

    Measured: 2 years

  2. Open Label Systemic Treatment Because of Inadequate Response to Primary Therapy

    Administration of any systemic therapy other than the immunosuppressive agents used for initial treatment, because of persistent or progressive chronic graft-versus-host disease

    Measured: 2 years

  3. Bronchiolitis Obliterans

    Development of bronchiolitis obliterans during treatment

    Measured: within 4 years

  4. Recurrent Malignancy

    Development of recurrent malignancy after enrollment in the study

    Measured: within 4 years

  5. Non-relapse Mortality

    Death without prior development of recurrent malignancy

    Measured: within 4 years

  6. Death or Recurrent Malignancy

    Death due to any cause or development of recurrent malignancy at any time after enrollment

    Measured: within 4 years

  7. Death

    Death from any cause after enrollment in the study

    Measured: within 4 years

  8. Withdrawal of Prednisone

    Withdrawal of treatment with prednisone after improvement or resolution of chronic GVHD

    Measured: within 4 years

  9. End of Systemic Treatment

    Withdrawal of all immunosuppressive treatment without recurrent malignancy

    Measured: within 4 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Aug 2004 (estimated)
  2. Study startMay 2004
  3. Primary completionJul 2008
  4. Study completionSep 2008

Why it stopped: Low probability of positive outcome

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
151 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00089141
Sponsor's protocol ID
1697.00
Other IDs
FHCRC-1697.00, ROCHE-FHCRC-1697.00, UMN-2004UC007, CDR0000378054
Status last verified
Aug 2009
First submitted
4 Aug 2004
Last updated
3 May 2013
Data monitoring committee
Yes

Publications

  • 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.

    PubMed 19270260

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing mycophenolate mofetil in people with Cancer, run by Martin, Paul at 16 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 3 May 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 16 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 26 Aug 2009). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Cure of Chronic GVHD Without Resorting to Secondary Systemic Therapy (time frame: 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00089141, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 May 2013.

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