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Thymoglobulin, Sirolimus and Mycophenolate Mofetil for Prevention of Acute Graft-Versus-Host Disease (GVHD)

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: NCT00506948
Recruitment Status : Terminated (Halted due to high incidence of veno-oclusive disease of the liver.)
First Posted : July 25, 2007
Results First Posted : February 20, 2014
Last Update Posted : February 20, 2014
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:

The goal of this clinical research study is to learn if the combination of rabbit anti-thymocyte globulin (Thymoglobulin®), sirolimus (Rapamune®), and mycophenolate mofetil (Cellcept®) can help to prevent graft versus host disease (GVHD). The safety of this drug combination will also be studied.

Primary Objective: To determine efficacy and toxicity of a regimen of thymoglobulin, sirolimus and mycophenolate mofetil for prevention of acute GVHD after allogeneic stem cell transplantation from human leukocyte antigen (HLA) identical related or unrelated donors.

Secondary Objective: To assess engraftment, chronic GVHD, relapse and survival.

Condition or disease Intervention/treatment Phase
Hematological Malignancies Myelodysplastic Syndrome Leukemia Lymphoma Drug: Mycophenolate Mofetil (MMF) Drug: Thymoglobulin Drug: Sirolimus Procedure: Stem Cell Transplant Phase 2

Detailed Description:

Rabbit anti-thymocyte globulin (rATG), sirolimus, and mycophenolate mofetil (MMF) are all designed to prevent GVHD.

If you are found to be eligible to take part in this study, you will receive rATG through a needle in your vein over 3-4 hours on each of the 4 days before the stem cell transplant.

Beginning 2 days before the transplant, you will take sirolimus by mouth once per day. You will continue to receive sirolimus until 90 days after the transplant. Beginning on Day 60, you will start taking increasingly lower doses of the study drug. This is done so you can taper down slowly, and be off of the drug on Day 90.

Beginning on the day of the transplant, you will take MMF by mouth 2 times a day. You will continue to take MMF until 27 days after the transplant.

Every week (for the first 90-100 days after the transplant) you will have study visits. At this visit, you will have a physical exam. Blood (about 1-2 tablespoons) will be drawn for routine tests.

You will remain on study for up to 90 days after transplantation. You will be taken off study if intolerable side effects occur.

Starting on Day 90 after the transplant, you will continue to follow up with your transplant doctor at least every 3 months through 1 year after the transplant. During these visits you will have physical exams. Blood (about 1-2 tablespoons) will be drawn for routine tests. Your doctor may request additional testing.

This is an investigational study. RATG, sirolimus, and MMF are all FDA approved for their use in the transplantation of solid organs (like kidney and liver). All 3 drugs are commercially available. This particular combination and dose schedule is considered investigational. Up to 30 patients will take part in this study. All will be enrolled at M.D. Anderson.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Thymoglobulin, Sirolimus and Mycophenolate Mofetil for Prevention of Acute GVHD Following Allogeneic Hematopoietic Stem Cell Transplantation
Study Start Date : September 2006
Actual Primary Completion Date : October 2010
Actual Study Completion Date : October 2010

Arm Intervention/treatment
Experimental: Thymoglobulin + Sirolimus + MMF
Thymoglobulin 1.5 mg/kg intravenous (IV) days -4, -3, -2, -1 before Stem Cell Transplant (Day 0); Sirolimus 6 mg IV on day -2 followed by 2 mg daily to maintain therapeutic levels and Mycophenolate Mofetil (MMF) 15 mg/kg IV or orally every 12 hours starting on day 0 until day+27.
Drug: Mycophenolate Mofetil (MMF)
15 mg/kg by vein or by mouth every 12 hours.
Other Names:
  • CellCept
  • MMF

Drug: Thymoglobulin
1.5 mg/kg by vein daily for 4 days
Other Names:
  • Antithymocyte Globulin
  • rATG
  • ATG

Drug: Sirolimus
6 mg daily by mouth for 1 day, followed by 2 mg daily for 1 day.
Other Name: Rapamune

Procedure: Stem Cell Transplant
Stem cell infusion on Day 0.
Other Names:
  • hematopoietic stem cell transplantation
  • HSCT
  • transplantation

Primary Outcome Measures :
  1. Failure Rate [ Time Frame: Baseline to 100 days post transplant ]
    Efficacy failure defined as a participants who had either grade 3-4 acute graft-versus-host disease (aGVHD) or treatment related mortality (TRM) within 100 days post transplant. Failure Rate calculated as (# of failures) / (# participants evaluated). Physical exam and bloodwork every week (for the first 90-100 days after the transplant).

  2. Number of Participants With Acute Graft-versus-host Disease (aGVHD) [ Time Frame: Baseline to 100 days post transplant ]
    Participants who had acute graft-versus-host disease (aGVHD) within 100 days post transplant. Physical exam and bloodwork every week (for the first 90-100 days after the transplant).

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.

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Patients with high risk hematological malignancies, including those with induction failure and after treated or untreated relapse. High risk hematological malignancies include: Acute myelogenous or lymphocytic leukemia with induction failure of after relapse, myelodysplastic syndrome of intermediate and high risk according to Greenberg criteria, chronic myelogenous leukemia in accelerated phase or blast crisis, non-Hodgkin's and Hodgkin's lymphoma with induction failure or relapse after chemotherapy and refractory or relapsed chronic lymphocytic leukemia.
  2. HLA-identical sibling or matched unrelated donor transplants not eligible for protocols of higher priority.
  3. Age 18-75 years.
  4. Bilirubin </=1.5 mg/dl, serum glutamic-pyruvic transaminase (SGPT) </= 200 IU/ml.
  5. Creatinine </=1.6 mg/dl.

Exclusion Criteria:

  1. Regimens including rituximab or alemtuzumab in the preparative regimen.
  2. Patients can not have received prior treatment with gemtuzumab.
  3. Planned conditioning chemotherapy for transplant can not include gemtuzumab.
  4. Planned conditioning chemotherapy for transplant can not include the busulfan and cyclophosphamide regimen.
  5. HIV seropositivity
  6. Uncontrolled infection, not responding to adequate antimicrobial therapy after 7 days of treatment. The protocol PI is the final arbiter of eligibility.
  7. Pregnancy
  8. Inability to sign consent.
  9. Patients who are past recipients of allogeneic or autologous stem cell transplants from any source.

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): NCT00506948

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United States, Texas
UT MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Genzyme, a Sanofi Company
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Principal Investigator: Amin Alousi, MD M.D. Anderson Cancer Center
Additional Information:
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Responsible Party: M.D. Anderson Cancer Center Identifier: NCT00506948    
Other Study ID Numbers: 2006-0435
First Posted: July 25, 2007    Key Record Dates
Results First Posted: February 20, 2014
Last Update Posted: February 20, 2014
Last Verified: January 2014
Keywords provided by M.D. Anderson Cancer Center:
Hematological Malignancies
Myelodysplastic Syndrome
Graft Versus Host Disease
Allogeneic Hematopoietic Stem Cell Transplantation
Rabbit Antithymocyte Globulin
Mycophenolate Mofetil
Additional relevant MeSH terms:
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Hematologic Neoplasms
Myelodysplastic Syndromes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms by Site
Mycophenolic Acid
Antilymphocyte Serum
Antibiotics, Antineoplastic
Antineoplastic Agents
Antibiotics, Antitubercular
Antitubercular Agents
Anti-Bacterial Agents
Anti-Infective Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs