Study of Unrelated Cord Blood Transplantation Using Tacrolimus and Sirolimus

July 22, 2016 updated by: Karen Ballen, Massachusetts General Hospital

Phase II Study of Sequential Unrelated Cord Blood Transplantation Using Tacrolimus and Sirolimus as Graft Versus Host Disease Prophylaxis

The purpose of this study is to measure the effectiveness of 2 drugs, tacrolimus and sirolimus, in preventing graft versus host disease (GVHD) after treatment with chemotherapy followed by donor cord blood transplantation.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

  • The chemotherapy portion of the study involves the intravenous administration of fludarabine, for six days (Days 8, 7, 6, 5,4, and 3) before transplant, melphalan, for one day (Day 2) before transplant. Antithymocyte globulin, or thymoglobulin, will be given IV daily for 4 days (days 7, 5, 3, and 1 before transplant). This drug also helps to suppress the immune system, allowing the cord blood cells to grow and reproduce.
  • Immunosuppression therapy consists of the drugs tacrolimus and sirolimus. The patient will receive these 3 days before the transplant and every day for 3-6 months after transplant. After the first 100 days post transplant, the doses of tacrolimus and sirolimus will begin to be reduced with the goal of having the patient off both drugs by 6-9 months after transplant.
  • After completion of conditioning therapy described above, the patient will receive 2 cord blood units 1-6 hours apart. To help with engraftment, the patient will also receive G-CSF starting on day five after transplant, until the patients white blood cells recover.
  • Follow-up visits will continue every 6 months after the last treatment dose and will last up to 2 years.
  • Blood tests will be drawn frequently to test whether the donor's immune cells have engrafted as well as to test the levels of Tacrolimus and Sirolimus.

Study Type

Interventional

Enrollment (Anticipated)

32

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Dana-Farber Cancer Institute
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Patients with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate
  • Non-Hodgkin's lymphoma, or Hodgkin's lymphoma: in Complete Remission >2 (second complete remission, third complete remission, etc) or in partial remission
  • Multiple myeloma: relapsed
  • Chronic lymphocytic leukemia, Rai stage III or IV, or lymphocyte doubling time of 6 months, or stage I-II, having progressed after > 2 chemotherapy regimens, in partial remission.
  • Acute myelogenous or lymphoblastic leukemia in second or subsequent remission or in first remission with adverse cytogenetic or antecedent hematologic disorder
  • Chronic myelogenous leukemia in accelerated or second stable phase, or imatinib resistant and not eligible for an ablative transplant
  • Myelodysplasia, previously treated or not eligible for ablative transplant
  • Age 18-65 years.
  • ECOG performance status of 0, 1, or 2.
  • Lack of 6/6 or 5/6 HLA-matched related, 10/10 matched unrelated donor, or unrelated donor not available within the time frame necessary to perform a potentially curative stem cell transplant.

Exclusion Criteria:

  • Cardiac disease:

    • symptomatic congestive heart failure or
    • radionuclide ventriculogram (RVG) or echocardiogram determined left ventricular ejection fraction of < 40%,
    • active angina pectoris, or
    • uncontrolled hypertension.

  • Pulmonary disease:

    • severe chronic obstructive lung disease, or
    • symptomatic restrictive lung disease, or
    • corrected DLCO of < 50% of predicted.

  • Renal disease:

    • serum creatinine > 2.0 mg/dl.

  • Hepatic disease:

    • serum bilirubin > 2.0 mg/dl (except in the case of Gilbert's syndrome or hemolytic anemia in which the bilirubin can be elevated greater than 2.0mg/dl),
    • SGOT or SGPT > 3 x normal.

  • Neurologic disease:

    • symptomatic leukoencephalopathy,
    • active central nervous system (CNS) malignancy or other neuropsychiatric abnormalities believed to preclude transplantation (previous CNS malignancy, presently in complete remission [CR] is not exclusion).
  • HIV antibody.
  • Uncontrolled infection.
  • Pregnancy or breast feeding mother.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To determine the effectiveness of tacrolimus and sirolimus in preventing graft versus host disease
Time Frame: 2 years
2 years

Secondary Outcome Measures

Outcome Measure
Time Frame
To evaluate the days to neutrophil engraftment and platelet engraftment
Time Frame: 2 years
2 years
To evaluate the relapse rate and overall disease free survival

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Massachusetts General Hospital

Collaborators

Dana-Farber Cancer Institute

Investigators

  • Principal Investigator: Karen K Ballen, MD, Massachusetts General Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2005

Primary Completion (ACTUAL)

November 1, 2007

Study Completion (ACTUAL)

November 1, 2011

Study Registration Dates

First Submitted

August 19, 2005

First Submitted That Met QC Criteria

August 22, 2005

First Posted (ESTIMATE)

August 23, 2005

Study Record Updates

Last Update Posted (ESTIMATE)

July 25, 2016

Last Update Submitted That Met QC Criteria

July 22, 2016

Last Verified

July 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 05-154

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Subscribe