Trial Search Results
Chronic Graft-versus-Host Disease Treatment (BMT CTN 0801)
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.
Stanford is currently not accepting patients for this trial.
Medical College of Wisconsin
Collaborator: National Marrow Donor Program
- Drug: Sirolimus + calcineurin inhibitor + prednisone
- Drug: Sirolimus + prednisone
Phase 2/Phase 3
- 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
- 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
- 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
- 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
- Known hypersensitivity to sirolimus.
Ages Eligible for Study
N/A - N/A
Genders Eligible for Study