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

Phase 2
18 Years
65 Years
Not Enrolling
Multiple Myeloma, Non-Hodgkin's Lymphoma, Hodgkin's Disease, Myelogenous Leukemia, Lymphoblastic Leukemia

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Trial Information

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

- 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

- 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.

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.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

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

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

Karen K Ballen, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Massachusetts General Hospital


United States: Institutional Review Board

Study ID:




Start Date:

August 2005

Completion Date:

November 2011

Related Keywords:

  • Multiple Myeloma
  • Non-Hodgkin's Lymphoma
  • Hodgkin's Disease
  • Myelogenous Leukemia
  • Lymphoblastic Leukemia
  • Hematologic malignancy
  • Cord blood transfusion
  • Graft versus host disease
  • tacrolimus
  • sirolimus
  • Graft vs Host Disease
  • Hodgkin Disease
  • Leukemia
  • Leukemia, Lymphoid
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Leukemia, Myeloid
  • Lymphoma
  • Lymphoma, Non-Hodgkin
  • Multiple Myeloma
  • Neoplasms, Plasma Cell



Dana-Farber Cancer Institute Boston, Massachusetts  02115
Massachusetts General Hospital Boston, Massachusetts  02114-2617