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A Phase II Trial of Sirolimus as Treatment of Steroid-Refractory or Steroid-Dependent Chronic Graft-Versus-Host Disease

Phase 2
13 Years
Not Enrolling
Graft vs Host Disease, Blood and Marrow Transplant (BMT)

Thank you

Trial Information

A Phase II Trial of Sirolimus as Treatment of Steroid-Refractory or Steroid-Dependent Chronic Graft-Versus-Host Disease

The purpose of this trial is to study the effectiveness of an immunosuppressive drug,
sirolimus, in the treatment of chronic graft versus host disease in combination with
prednisone. Graft versus host disease (GVHD) is a common complication in patients who have
received blood or marrow transplantation from a related or unrelated donor. Chronic GVHD
occurs approximately 100 days after transplantation and is the result of the donor immune
system recognizing the patient's tissues as foreign and creating harmful effects on the
patient';s organs. We hope the use of sirolimus will decrease the significant disabling
effects and deaths caused by chronic GVHD.

Inclusion Criteria:

- Histologically-confirmed active chronic GVHD >100 days following
allogeneic bone marrow/peripheral blood/umbilical cord blood transplantation that has
failed prior therapy. In the event that histological confirmation poses undue risk,
clinical evaluation is sufficient.

- Women must have a negative pregnancy test before sirolimus administration and women
of child-bearing potential agree to use a medically acceptable contraceptive
throughout the treatment period and for 3 months after discontinuation of sirolimus.
Any woman becoming pregnant during the treatment period must discontinue the use of

- Absolute neutrophil count (ANC) >1000/mm^3, unless receiving G-CSF to maintain
neutrophil count >500/mm^3

- Discontinuation of cyclosporine or FK506 at the time of initiating sirolimus with
cyclosporine trough level <100 mg/dl and FK506 level < 5 mg/dl

- Karnofsky performance score > or = 50 during pre-study screening

- Written, signed, and dated informed consent

Exclusion Criteria:- Uncontrolled systemic infection

- Unstable disease states (i.e., hepatic failure, ventilatory-dependent respiratory
failure, etc.)

- Serum creatinine > or = 3.0 mg/dL, platelet count < or = 50,000/mm^3

- History of Post-transplant microangiopathic hemolytic anemia

- Uncontrolled hyperlipidemia

- Use of any investigational drug within 4 weeks of entry into the study

- Use of methotrexate or antibody therapies within 24 hours of sirolimus administration

- Inability to tolerate oral therapy for any reason

- Evidence of infiltrate, cavitation, or consolidation on chest x-ray during pre-study

- Known hypersensitivity to macrolide antibiotics

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Clinical activity will be monitored at 3 month intervals after the initiation of sirolimus until 2 years after the initiation of sirolimus

Outcome Time Frame:

every 3 months until 2 years after enrollment

Safety Issue:


Principal Investigator

Laura Johnston

Investigator Role:

Principal Investigator

Investigator Affiliation:

Stanford University


United States: Institutional Review Board

Study ID:




Start Date:

November 2005

Completion Date:

October 2012

Related Keywords:

  • Graft Vs Host Disease
  • Blood and Marrow Transplant (BMT)
  • GVHD
  • Graft vs Host Disease



Stanford University School of Medicine Stanford, California  94305-5317