Working… Menu

Tacrolimus and Sirolimus as Prophylaxis After Allogenic Non-myeloablative Peripheral Blood 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: NCT00282282
Recruitment Status : Completed
First Posted : January 26, 2006
Results First Posted : April 8, 2014
Last Update Posted : May 12, 2014
Brigham and Women's Hospital
Information provided by (Responsible Party):
Vincent T. Ho, MD, Dana-Farber Cancer Institute

Brief Summary:
The purpose of this study is to extend the use of Tacrolimus and Sirolimus to determine how effective it is in preventing graft versus host disease (GVHD)in patients that have received non-myeloablative peripheral blood stem cell transplantation.

Condition or disease Intervention/treatment Phase
Graft Versus Host Disease GVHD Drug: tacrolimus Drug: sirolimus Drug: fludarabine Drug: busulfex Phase 2

Detailed Description:
  • After the screening procedures confirm that the patient is eligible to participate in the research study, they will be admitted to the hospital to receive chemotherapy and stem cell transplantation (SCT). The duration of the hospitalization for the procedure is approximately 8 days.
  • Patients will receive fludarabine once daily over 30 minutes intravenously for 4 days and busulfex once daily over 3 hours intravenously each day for the same 4 days.
  • Just prior to the transplant and following the transplant the patient will receive sirolimus and tacrolimus to help prevent Graft versus Host Disease (GvHD). Both medications are taken orally.
  • Patients will also take medications to help prevent possible infections (e.g. acyclovir). Filgrastim, a white blood cell growth factor, will be given daily in an injection under the skin, starting the day after the stem cell transplant and until the patients blood counts have recovered.
  • After the stem cell infusion, the patient will be examined and have blood tests weekly for 1 month. At about the 1-month visit, a bone marrow biopsy and/or blood tests will be performed to determine the percentage of donor's cells in the blood or bone marrow. These tests will be repeated at 3-4 months after transplant.
  • At 3-4 months after the transplant, patients will also have tests to reassess the response of your disease to transplant. This may involve a bone marrow biopsy, blood tests, and/or radiology studies depending upon the type of cancer.
  • Follow-up will continue for the remainder of the patients life.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 31 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Tacrolimus and Sirolimus as Graft Versus Host Disease Prophylaxis After Allogeneic Non-myeloablative Peripheral Blood Stem Cell Transplantation
Study Start Date : January 2006
Actual Primary Completion Date : January 2009
Actual Study Completion Date : July 2009

Resource links provided by the National Library of Medicine

Intervention Details:
  • Drug: tacrolimus
    Given orally just prior to and following stem cell transplant
  • Drug: sirolimus
    Given orally just prior to and following stem cell transplant
  • Drug: fludarabine
    Given once daily over 30 minutes for 4 days
  • Drug: busulfex
    Given intravenously over 3 hours for 4 days

Primary Outcome Measures :
  1. Incidence of Grade II-IV Acute GVHD (aGVHD) Developing by Day 100 Following Non-myeloablative PBSC Transplantation Using Tacrolimus and Sirolimus. [ Time Frame: 100 days ]
    All participants received tacrolimus and sirolimus in this one arm study. There were no participants considered unevaluable for this measure (deceased prior to day 100). The total number of people who developed grade II-IV aGVHD before day 100 are reported here.

Secondary Outcome Measures :
  1. Percentage of Participants With ≥90 Percent Donor-derived Hematopoeisis Around 100 Days Post Transplantation [ Time Frame: 100 days ]
    The percentage of participants with ≥90 percent donor-derived hematopoeisis was assessed around day +100 using peripheral blood chimerism.

  2. Disease Response. [ Time Frame: 2 years ]
    Disease response was assessed as 2 year progression-free survival. The median follow-up time was 1.84 years. The percentage of participants with who reached this timepoint with no disease progression are reported.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
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 are at a high risk of complications after conventional transplantation
  • Availability of a related donor who is identical at 6 HLA loci
  • Greater than 18 years of age
  • Performance status 0-2
  • Life expectancy of > 100 days

Exclusion Criteria:

  • Pregnancy
  • Evidence of HIV infection
  • Heart failure uncontrolled medication
  • Total bilirubin > 2.0mg/dl that is due to hepatocellular dysfunction
  • AST >90
  • Serum Creatinine >2.0
  • Cholesterol > 300mg/dl while adequately treated

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): NCT00282282

Layout table for location information
United States, Massachusetts
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Dana-Farber Cancer Institute
Brigham and Women's Hospital
Layout table for investigator information
Principal Investigator: Vincent Ho, MD Dana-Farber Cancer Institute
Publications of Results:
Layout table for additonal information
Responsible Party: Vincent T. Ho, MD, Principal Investigator, Dana-Farber Cancer Institute Identifier: NCT00282282    
Other Study ID Numbers: 05-362
First Posted: January 26, 2006    Key Record Dates
Results First Posted: April 8, 2014
Last Update Posted: May 12, 2014
Last Verified: March 2014
Keywords provided by Vincent T. Ho, MD, Dana-Farber Cancer Institute:
non-myeloablative peripheral blood stem cell
Additional relevant MeSH terms:
Layout table for MeSH terms
Graft vs Host Disease
Immune System Diseases
Antineoplastic Agents
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Calcineurin Inhibitors
Enzyme Inhibitors
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents
Alkylating Agents
Antineoplastic Agents, Alkylating
Myeloablative Agonists