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Chronic Graft-versus-Host Disease Treatment (BMT CTN 0801)
Not Recruiting
Trial ID: NCT01106833
Purpose
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.
Official Title
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)
Stanford Investigator(s)
Laura Johnston
Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)
Robert Negrin
Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)
Wen-Kai Weng, MD, PhD
Associate Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy) and, by courtesy, of Dermatology
Robert Lowsky
Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)
Sally Arai
Associate Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)
Judith Shizuru
Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy) and of Pediatrics (Stem Cell Transplantation)
Eligibility
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Intervention(s):
drug: Sirolimus + calcineurin inhibitor + prednisone
drug: Sirolimus + prednisone
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Physician Referrals
650-723-0822