Sirolimus With Tacrolimus for Graft-vs-Host Disease Prophylaxis After Related Stem Cell Transplantation

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00144703
Recruitment Status : Completed
First Posted : September 5, 2005
Last Update Posted : January 26, 2012
Brigham and Women's Hospital
Information provided by (Responsible Party):
Corey S. Cutler, MD, MPH, Dana-Farber Cancer Institute

Brief Summary:
The purpose of this study is to evaluate the ability of sirolimus to prevent graft versus host disease (GVHD) in patients following stem cell transplant from a related donor.

Condition or disease Intervention/treatment Phase
Hematologic Malignancies Myelogenous Leukemia Acute Lymphoblastic Leukemia Myelodysplastic Syndrome Non-Hodgkin's Lymphoma Hodgkin's Disease Drug: Sirolimus Drug: Tacrolimus Phase 2

Detailed Description:
  • Therapy to prevent GVHD will consist of an infusion of tacrolimus intravenously and sirolimus orally once per day starting 3 days before stem cell infusion. This will take place in the hospital where the patient will remain for the duration of the transplant.
  • Sirolimus will continue for 9 weeks at at stable dose, then will be tapered by 1/3 on week 9 and 1/3 on week 17. It will be stopped on week 26 if there is no significant evidence of GVHD. These dose modifications will occur at home and patients will be seen weekly for the first 2 months after discharge.
  • If GVHD is present, the tapering schedule wil be slower and based upon the patient's clinical condition.
  • Tacrolimus will be given orally once the patient is discharged from the hospital and will be tapered at the same schedule as sirolimus. Blood levels of both tacrolimus and sirolimus will be monitored and the dose adjusted accordingly.
  • During the year following stem cell transplant, blood work will be performed to evaluate the immune system and GVHD.

Study Type : Interventional  (Clinical Trial)
Enrollment : 55 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Open Label Phase II Trial of Sirolimus in Combination With Tacrolimus for Graft-vs-Host Disease Prophylaxis After Matched, Related Allogeneic Hematopoietic Stem Cell Transplantation
Study Start Date : July 2002
Actual Primary Completion Date : November 2006
Actual Study Completion Date : November 2006

Primary Outcome Measures :
  1. To determine the feasibility of using sirolimus and tacrolimus without methotrexate for GVHD prophylaxis after stem cell transplantation.

Secondary Outcome Measures :
  1. To compare the rates of grade II-IV and III-IV acute GVHD with historical controls
  2. to determine the incidence of 100 day mortality after stem cell transplant using this regimen
  3. to determine the overall survival at 1 year after transplantation in this patient population.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients with hematologic malignancies who will undergo allogeneic HSCT from matched family-members.
  • Age greater than 18
  • ECOG performance status 0-2
  • Total bilirubin < 2.0 mg/dl
  • AST < 90 IU
  • Serum creatinine < 2.0 mg/dl

Exclusion Criteria:

  • Active, uncontrolled infection
  • Ejection fraction < 45% by echocardiogram or MUGA scan
  • Forced vital capacity < 60%
  • Uncontrolled hypertension
  • Second transplantation
  • Evidence of HIV infection
  • Cholesterol > 300 mg/dl
  • Relapsed aggressive Burkitt's or Burkitt's-like lymphoma

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00144703

United States, Massachusetts
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Dana-Farber Cancer Institute
Brigham and Women's Hospital
Principal Investigator: Corey Cutler, MD, MPH Dana-Farber Cancer Institute

Responsible Party: Corey S. Cutler, MD, MPH, Principal Investigator, Dana-Farber Cancer Institute Identifier: NCT00144703     History of Changes
Other Study ID Numbers: 02-090
First Posted: September 5, 2005    Key Record Dates
Last Update Posted: January 26, 2012
Last Verified: January 2009

Keywords provided by Corey S. Cutler, MD, MPH, Dana-Farber Cancer Institute:
Graft versus host disease
Stem cell transplantation

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Lymphoma, Non-Hodgkin
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphoid
Leukemia, Myeloid
Hodgkin Disease
Graft vs Host Disease
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Calcineurin Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Anti-Bacterial Agents
Anti-Infective Agents