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

This study has been completed.
Brigham and Women's Hospital
Information provided by (Responsible Party):
Corey S. Cutler, MD, MPH, Dana-Farber Cancer Institute Identifier:
First received: September 1, 2005
Last updated: January 24, 2012
Last verified: January 2009
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 Intervention Phase
Hematologic Malignancies
Myelogenous Leukemia
Acute Lymphoblastic Leukemia
Myelodysplastic Syndrome
Non-Hodgkin's Lymphoma
Hodgkin's Disease
Drug: Sirolimus
Drug: Tacrolimus
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:

Further study details as provided by Dana-Farber Cancer Institute:

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

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

Estimated Enrollment: 55
Study Start Date: July 2002
Study Completion Date: November 2006
Primary Completion Date: November 2006 (Final data collection date for primary outcome measure)
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.

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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
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Please refer to this study by its identifier: 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
  More Information

Responsible Party: Corey S. Cutler, MD, MPH, Principal Investigator, Dana-Farber Cancer Institute Identifier: NCT00144703     History of Changes
Other Study ID Numbers: 02-090
Study First Received: September 1, 2005
Last Updated: January 24, 2012

Keywords provided by 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
Graft vs Host Disease
Hodgkin 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 processed this record on April 24, 2017