Clinical trial · Interventional
Mycophenolate Mofetil (MMF) for Treatment of Chronic Graft-versus-host Disease (GVHD)
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
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): Low probability of positive outcome
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Cancer | Malignant Neoplasm | ALIAS | 0.90 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| mycophenolate mofetil | Drug | — | UNRESOLVED |
| placebo | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- ACTIVE_COMPARATOR
- label
- Mycophenolate mofetil
- description
- Patients receive oral mycophenolate mofetil twice daily.
- interventionNames
- Drug: mycophenolate mofetil
- type
- PLACEBO_COMPARATOR
- label
- Placebo
- description
- Patients receive oral placebo twice daily
- interventionNames
- Drug: placebo
Primary outcomes (1)
- measure
- Cure of Chronic GVHD Without Resorting to Secondary Systemic Therapy
- timeFrame
- 2 years
- description
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 4 Years
Show eligibility criteria text
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
References
Publications (1)
- RESULTMartin 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 19270260