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

M

Martin, Paul

Status and phase

Terminated
Phase 3

Conditions

Cancer

Treatments

Drug: mycophenolate mofetil
Drug: placebo

Study type

Interventional

Funder types

Other
NIH

Identifiers

NCT00089141
1697.00
ROCHE-FHCRC-1697.00
CDR0000378054 (Registry Identifier)
FHCRC-1697.00
UMN-2004UC007

Details and patient eligibility

About

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.

Full 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.

Enrollment

151 patients

Sex

All

Ages

4+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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

Trial design

151 participants in 2 patient groups, including a placebo group

Mycophenolate mofetil
Active Comparator group
Description:
Patients receive oral mycophenolate mofetil twice daily.
Treatment:
Drug: mycophenolate mofetil
Placebo
Placebo Comparator group
Description:
Patients receive oral placebo twice daily
Treatment:
Drug: placebo

Trial contacts and locations

0

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Data sourced from clinicaltrials.gov

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