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



Status:Completed
Conditions:Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:Any
Updated:12/7/2018
Start Date:April 2010
End Date:June 2018

Use our guide to learn which trials are right for you!

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)

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.

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

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 initial therapy for
chronic GVHD, or before their 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 two arms.

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.
We found this trial at
31
sites
201 Dowman Dr
Atlanta, Georgia 30303
(404) 727-6123
Emory University Emory University, recognized internationally for its outstanding liberal artscolleges, graduate and professional schools,...
?
mi
from
Atlanta, GA
Click here to add this to my saved trials
3400 N Charles St
Baltimore, Maryland 21205
410-516-8000
Johns Hopkins University The Johns Hopkins University opened in 1876, with the inauguration of its...
?
mi
from
Baltimore, MD
Click here to add this to my saved trials
666 Elm Street
Buffalo, New York 14263
(716) 845-2300
Roswell Park Cancer Institute Welcome to Roswell Park Cancer Institute (RPCI), America's first cancer center...
?
mi
from
Buffalo, NY
Click here to add this to my saved trials
5801 South Ellis Avenue
Chicago, Illinois 60637
 773.702.1234
University of Chicago One of the world's premier academic and research institutions, the University of...
?
mi
from
Chicago, IL
Click here to add this to my saved trials
1600 SW Archer Rd # M509
Gainesville, Florida 32610
?
mi
from
Gainesville, FL
Click here to add this to my saved trials
Minneapolis, Minnesota 55455
(612) 625-5000
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
?
mi
from
Minneapolis, MN
Click here to add this to my saved trials
1100 Fairview Avenue North
Seattle, Washington 98109
(206) 667-5000
Fred Hutchinson Cancer Research Center At Fred Hutchinson Cancer Research Center, our interdisciplinary teams of...
?
mi
from
Seattle, WA
Click here to add this to my saved trials
Ann Arbor, Michigan 48109
?
mi
from
Ann Arbor, MI
Click here to add this to my saved trials
?
mi
from
Atlanta, GA
Click here to add this to my saved trials
101 Manning Dr
Chapel Hill, North Carolina 27599
(919) 966-4131
University of North Carolina Hospital at Chapel Hill The UNC Health Care System is a...
?
mi
from
Chapel Hill, NC
Click here to add this to my saved trials
171 Ashley Avenue
Charleston, South Carolina 29425
843-792-1414
Medical University of South Carolina The Medical University of South Carolina (MUSC) has grown from...
?
mi
from
Charleston, SC
Click here to add this to my saved trials
Cincinnati, Ohio 45236
?
mi
from
Cincinnati, OH
Click here to add this to my saved trials
?
mi
from
Cleveland, OH
Click here to add this to my saved trials
1500 East Duarte Road
Duarte, California 91010
626-256-HOPE (4673)
City of Hope National Medical Center City of Hope is dedicated to making a difference...
?
mi
from
Duarte, CA
Click here to add this to my saved trials
2301 Erwin Rd
Durham, North Carolina 27710
919-684-8111
Duke Univ Med Ctr As a world-class academic and health care system, Duke Medicine strives...
?
mi
from
Durham, NC
Click here to add this to my saved trials
30 Prospect Ave
Hackensack, New Jersey 07601
(201) 996-2000
Hackensack University Medical Center Hackensack University Medical Center, part of the Hackensack University Health Network,...
?
mi
from
Hackensack, NJ
Click here to add this to my saved trials
?
mi
from
Houston, TX
Click here to add this to my saved trials
Kansas City, Kansas 66160
?
mi
from
Kansas City, KS
Click here to add this to my saved trials
?
mi
from
La Jolla, CA
Click here to add this to my saved trials
600 Highland Ave
Madison, Wisconsin 53792
(608) 263-6400
University of Wisconsin Hospital and Clinics UW Health strives to meet the health needs of...
?
mi
from
Madison, WI
Click here to add this to my saved trials
1275 York Ave
New York, New York 10021
(212) 639-2000
Memorial Sloan Kettering Cancer Center Memorial Sloan Kettering Cancer Center — the world's oldest and...
?
mi
from
New York, NY
Click here to add this to my saved trials
Oklahoma City, Oklahoma 73104
?
mi
from
Oklahoma City, OK
Click here to add this to my saved trials
Emile St
Omaha, Nebraska 68198
(402) 559-4000
Univ of Nebraska Med Ctr A vital enterprise in the nation’s heartland, the University of...
?
mi
from
Omaha, NE
Click here to add this to my saved trials
Philadelphia, Pennsylvania 19104
?
mi
from
Philadelphia, PA
Click here to add this to my saved trials
4800 Friendship Ave
Pittsburgh, Pennsylvania 15224
(412) 578-5000
Western Pennsylvania Hospital Featuring 308 private patient beds, West Penn Hospital has served Bloomfield and...
?
mi
from
Pittsburgh, PA
Click here to add this to my saved trials
?
mi
from
Portland, OR
Click here to add this to my saved trials
?
mi
from
Richmond, VA
Click here to add this to my saved trials
Rochester, New York 55905
?
mi
from
Rochester, NY
Click here to add this to my saved trials
Saint Louis, Missouri 63110
?
mi
from
Saint Louis, MO
Click here to add this to my saved trials
San Antonio, Texas 78229
?
mi
from
San Antonio, TX
Click here to add this to my saved trials
Stanford, California 94305
?
mi
from
Stanford, CA
Click here to add this to my saved trials