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)

Wen-Kai Weng, MD, PhD
Wen-Kai Weng, MD, PhD

Associate Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy) and, by courtesy, of Dermatology

Robert Lowsky
Robert Lowsky

Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)

Laura Johnston
Laura Johnston

Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)

Robert Negrin
Robert Negrin

Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)

Sally Arai
Sally Arai

Associate Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)

Judith Shizuru
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

New Trial Alerts

Receive email alerts when trials open to patients.