Chronic Graft-versus-Host Disease Treatment (BMT CTN 0801)

2014-08-27 03:14:28 | BioPortfolio


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.


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.

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment


Chronic GVHD


Sirolimus + calcineurin inhibitor + prednisone, Sirolimus + prednisone, Sirolimus + extracorporeal photopheresis + prednisone


University of Alabama at Birmingham
United States




National Heart, Lung, and Blood Institute (NHLBI)

Results (where available)

View Results


Published on BioPortfolio: 2014-08-27T03:14:28-0400

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Medical and Biotech [MESH] Definitions

A macrolide compound obtained from Streptomyces hygroscopicus that acts by selectively blocking the transcriptional activation of cytokines thereby inhibiting cytokine production. It is bioactive only when bound to IMMUNOPHILINS. Sirolimus is a potent immunosuppressant and possesses both antifungal and antineoplastic properties.

A derivative of sirolimus and an inhibitor of TOR SERINE-THREONINE KINASES. It is used to prevent GRAFT REJECTION in heart and kidney transplant patients by blocking cell proliferation signals. It is also an ANTINEOPLASTIC AGENT.

A family of immunophilin proteins that bind to the immunosuppressive drugs TACROLIMUS (also known as FK506) and SIROLIMUS. EC 5.2.1.-

A 12-KDa tacrolimus binding protein that is found associated with and may modulate the function of calcium release channels. It is a peptidyl-prolyl cis/trans isomerase which is inhibited by both tacrolimus (commonly called FK506) and SIROLIMUS.

Members of a family of highly conserved proteins which are all cis-trans peptidyl-prolyl isomerases (PEPTIDYLPROLYL ISOMERASE). They bind the immunosuppressant drugs CYCLOSPORINE; TACROLIMUS and SIROLIMUS. They possess rotamase activity, which is inhibited by the immunosuppressant drugs that bind to them.

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