A Phase II/III Randomized, Multicenter Trial Comparing Sirolimus Plus Prednisone, Sirolimus/Extracorporeal Photopheresis Plus Prednisone, and Sirolimus/Calcineurin Inhibitor Plus Prednisone for the Treatment of Chronic Graft-versus-Host Disease (BMT CTN #

Status
Active
Cancer Type
Bone and Soft Tissue (including Sarcoma)
Trial Phase
Phase II
Phase III
Eligibility
Not specified, Male and Female
Study Type
Biomarker/Laboratory analysis
Supportive care
NCD ID
NCT01106833
Protocol IDs
609 (primary)
BMT CTN 0801
U01HL069294-06
Study Sponsor
National Heart, Lung, and Blood Institute

Summary

This study is designed as a combined Phase II/III, randomized, open label, multicenter, prospective comparative study of sirolimus plus prednisone, sirolimus/extracorporeal photopheresis 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 one of the two pre-specified experimental arms (Sirolimus + prednisone, Sirolimus + ECP + prednisone) or the comparator arm (Sirolimus + calcineurin inhibitor + prednisone) in a 1:1 ratio.

Objectives

Background: Chronic GVHD is a medical condition that can become very serious. Chronic GVHD is a common development after allogeneic transplant that occurs when the donor cells attack and damage tissues. The primary purpose of this study is to compare treatment regimens that contain sirolimus without a calcineurin inhibitor to a comparator regimen of sirolimus with a calcineurin inhibitor and evaluate how well chronic GVHD responds to treatment. The combinations of medications in this study are:

  • Sirolimus + calcineurin inhibitor + prednisone
  • Sirolimus + prednisone
  • Sirolimus + extracorporeal photopheresis + prednisone

The goal is to select a treatment regimen for further comparison in the Phase III trial.

Design Narrative: The intent is to enroll subjects at the start of their initial therapy for high-risk chronic GVHD, or before their standard-risk chronic GVHD is refractory to glucocorticoid therapy, or is chronically dependent upon glucocorticoid therapy and multiple secondary systemic immunosuppressive agents. Patients will be stratified by transplant center and will be randomized to one of the three arms.

Only one of the two Phase II studies above will proceed into the Phase III component of the study.

Treatment Sites in Georgia


Aflac Cancer and Blood Disorders Center of Children’s at Egleston
1405 Clifton Road NE
3rd Floor
Atlanta, GA 30322
404-785-0853
www.choa.org

Study Coordinator:
Sindy Midoro

Doctors:

Kuang-Yueh Chiang MD, PhD
Ann E. Haight MD
John T. Horan MD, MPH
Muna Qayed MD

Aflac Cancer and Blood Disorders Center of Children’s at Scottish Rite
5455 Meridian Mark Road
Suite 400
Atlanta, GA 30342
404-785-2215
www.choa.org

Study Coordinator:
Sindy Midoro

Doctors:

Kuang-Yueh Chiang MD, PhD
Ann E. Haight MD
John T. Horan MD, MPH

Winship Cancer Institute of Emory University
1365 Clifton Road NE
Building C
Atlanta, GA 30322
404-778-5180
winshipcancer.emory.edu

Study Coordinator:
Heather Renfroe
404-778-5127

Doctors:

Amelia A. Langston MD
Edmund K. Waller MD, PhD, FACP