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CompletedNCT00133367Updated Jul 25, 2016

Study of Unrelated Cord Blood Transplantation Using Tacrolimus and Sirolimus

A Phase 2 interventional study of Tacrolimus and Sirolimus in Multiple Myeloma, Non-Hodgkin's Lymphoma and Hodgkin's Disease, sponsored by Massachusetts General Hospital. Completed at 2 sites in United States. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2016-07-25.

Sponsored by Massachusetts General Hospital · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
32
Allocation
Non-randomized
Ages
18 Years to 65 Years
Sex
All
01

Study summary

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.

Read the 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.
02

Conditions studied

  • Multiple Myeloma
  • Non-Hodgkin's Lymphoma
  • Hodgkin's Disease
  • Myelogenous Leukemia
  • Lymphoblastic Leukemia

Keywords

  • Hematologic malignancy
  • Cord blood transfusion
  • Graft versus host disease
  • tacrolimus
  • sirolimus
03

In context

Leukemia

5,441 studies on the registry are indexed under Leukemia; 636 are open to participants now.

This study's planned enrollment of 32 is below the median of 38 across 4,247 interventional studies indexed under Leukemia.

Browse Leukemia studies →

Lead sponsor

Massachusetts General Hospital is the lead sponsor of 2,536 studies on the registry; 446 are open to participants now.

Of its 214 completed or terminated interventional studies of FDA-regulated products, 161 (75%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Single group
Masking
None (open label)
Enrollment
32 participants (estimated)

Interventions

  • DrugTacrolimus

    Given three days before transplant and every day for 3-6 months after transplant. After first 100 days post-transplant, the dose will be reduced.

  • DrugSirolimus

    Given three days before transplant and every day for 3-6 months after transplant. After first 100 days post-transplant, the dose will be reduced.

  • DrugG-CSF

    Given starting on day 5 after transplant until the subjects white blood cell count recovers.

  • DrugAntithymocyte globulin

    Given intravenously for 4 days before transplant (days 7, 5, 3, 1).

  • DrugThymoglobulin

    Given intravenously for 4 days before transplant (days 7, 5, 3, 1).

  • DrugFludarabine

    Given intravenously for six days prior to transplant (days 8,7,6,5,4,3).

  • DrugMelphalan

    Given intravenously on day 2 before transplant.

06

What researchers measure

Primary outcomes

  1. To determine the effectiveness of tacrolimus and sirolimus in preventing graft versus host disease

    Time frame: 2 years

Secondary outcomes

  1. To evaluate the days to neutrophil engraftment and platelet engraftment

    Time frame: 2 years

  2. To evaluate the relapse rate and overall disease free survival

07

Study locations

2 sites
  • Massachusetts General Hospital
    Boston, Massachusetts 02114, United States
  • Dana-Farber Cancer Institute
    Boston, Massachusetts 02115, United States
08

References and documents

Publications

  • Brown JA, Stevenson K, Kim HT, Cutler C, Ballen K, McDonough S, Reynolds C, Herrera M, Liney D, Ho V, Kao G, Armand P, Koreth J, Alyea E, McAfee S, Attar E, Dey B, Spitzer T, Soiffer R, Ritz J, Antin JH, Boussiotis VA. Clearance of CMV viremia and survival after double umbilical cord blood transplantation in adults depends on reconstitution of thymopoiesis. Blood. 2010 May 20;115(20):4111-9. doi: 10.1182/blood-2009-09-244145. Epub 2010 Jan 27. PubMed 20107229 ↗
09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 25, 2016, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT00133367
Lead sponsor
Massachusetts General Hospital
Collaborators
Dana-Farber Cancer Institute
Responsible party
Karen Ballen (Principal Investigator, Massachusetts General Hospital) — Principal investigator
First posted
Aug 23, 2005
Start date
Aug 2005
Primary completion
Nov 2007
Completion
Nov 2011
Last update
Jul 25, 2016

Study contacts

Karen K Ballen, MD
principal investigator · Massachusetts General Hospital

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jul 2016. You cannot join it, but the record below documents what was studied.

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