Chronic Graft-versus-Host Disease Treatment (BMT CTN 0801)
Trial ID or NCT#
This study is designed as a combined Phase II/III, randomized, open label, multicenter, prospective comparative study of sirolimus plus prednisone versus sirolimus/calcineurin-inhibitor plus prednisone for the treatment of chronic GVHD. Patients will be stratified by transplant center and will be randomized to an experimental arm of one of the two pre-specified experimental arms (sirolimus + prednisone or the comparator arm of sirolimus + calcineurin inhibitor + prednisone) in a 1:1 ratio.
A Phase II/III Randomized, Multicenter Trial Comparing Sirolimus Plus Prednisone and Sirolimus/Calcineurin Inhibitor Plus Prednisone for the Treatment of Chronic Graft-versus-Host Disease (BMT CTN Protocol #0801)
- - Suitable candidates are patients with classic chronic GVHD or overlap syndrome (classic chronic plus acute GVHD)that is: a)Previously untreated (newly diagnosed) as defined by having received < 14 days of prednisone (or equivalent) before enrollment/randomization to study therapy; b)Previously treated but inadequately responding after ≤ 16 weeks of initial therapy with prednisone and/or calcineurin inhibitor (CNI) ± additional non-sirolimus agent (started at the time of chronic GVHD diagnosis). - Patient or guardian willing and able to provide informed consent. - Stated willingness to use contraception in women of childbearing potential. - Stated willingness of patient to comply with study procedures and reporting requirements.
- - Patients with late persistent acute GVHD or recurrent acute GVHD only. - Inability to begin prednisone therapy at a dose of greater than 0.5 mg/kg/day. - Receiving sirolimus for treatment of chronic GVHD (sirolimus for prophylaxis or treatment of acute GVHD is acceptable). - Already receiving sirolimus (for prophylaxis or treatment of acute GVHD) with prednisone at ≥ 0.25 mg/kg/day (or equivalent) ± additional agents. - Receiving therapy for chronic GVHD for more than 16 weeks. - Invasive fungal or viral infection not responding to appropriate antifungal or antiviral therapies. - Inadequate renal function defined as measured creatinine clearance less than 50 mL/min/1.73 m^2 based on the Cockcroft-Gault formula (adults) or Schwartz formula (age less than or equal to 12 years). Adults: estimated creatinine clearance rate (eCCr) (mL/min/) = (140 - age) x mass (kg) x (0.85 if female)/72 x serum creatinine (mg/dL; Creatinine clearance (mL/min/1.73m^2) = eCCr x 1.73/Body Surface Area (BSA) (m^2); Children: eCCr (mL/min/1.73 m^2) = k x height (cm) / serum creatinine (mg/dL) k = 0.33 (pre-term), 0.45 (full term to 1 year old), 0.55 (age 1-12 years). - Inability to tolerate oral medications. - Absolute neutrophil count less than 1500 per microliter. - Requirement for platelet transfusions. - Pregnancy (positive serum β-HCG) or breastfeeding. - Receiving any treatment for persistent, progressive or recurrent malignancy. - Progressive or recurrent malignancy defined other than by quantitative molecular assays. - Known hypersensitivity to sirolimus.
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