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Halted due to high incidence of veno-oclusive disease of the liver.
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| Name | Class |
|---|---|
| Genzyme, a Sanofi Company | INDUSTRY |
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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.
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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Thymoglobulin + Sirolimus + MMF | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Mycophenolate Mofetil (MMF) | Drug | 15 mg/kg by vein or by mouth every 12 hours. |
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| Measure | Description | Time Frame |
|---|---|---|
| Failure Rate | 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). | Baseline to 100 days post transplant |
| Number of Participants With Acute Graft-versus-host Disease (aGVHD) | 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). | Baseline to 100 days post transplant |
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Inclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Amin Alousi, MD | M.D. Anderson Cancer Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UT MD Anderson Cancer Center | Houston | Texas | 77030 | United States |
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| Label | URL |
|---|---|
| University of Texas (UT) MD Anderson Cancer Center Official Website | View source |
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Of the 13 participants enrolled, three participants were ineligible and excluded from the study before treatment.
Recruitment Period: September 08, 2006 to October 27, 2009. All recruitment done in a medical clinic setting.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Thymoglobulin | Drug | 1.5 mg/kg by vein daily for 4 days |
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| Sirolimus | Drug | 6 mg daily by mouth for 1 day, followed by 2 mg daily for 1 day. |
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| Stem Cell Transplant | Procedure | Stem cell infusion on Day 0. |
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| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median | Full Range | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Failure Rate | 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). | Not Posted | Baseline to 100 days post transplant | Participants | |||||||||||||||||||||||||||||||
| Primary | Number of Participants With Acute Graft-versus-host Disease (aGVHD) | 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). | Posted | Number | participants | Baseline to 100 days post transplant |
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Adverse event reporting from active treatment completed on day 90 (last day of sirolimus) through post-study surveillance to Day +100. Adverse events were collected for a total study period of 2 years and 11 months.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. | 6 | 10 | 4 | 10 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Death | General disorders | CTCAE (3.0) | Systematic Assessment | Two deaths related to cases of veno-occlusive disease of the liver. |
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| Veno-occlusive disease of the liver | Renal and urinary disorders | CTCAE (3.0) | Systematic Assessment | Two of the four cases were fatal. |
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| Pneumonia | Infections and infestations | CTCAE (3.0) | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | CTCAE (3.0) | Systematic Assessment | Grade 2 |
|
| Hemorrhagic Cystitis | Renal and urinary disorders | CTCAE (3.0) | Systematic Assessment |
| |
| Veno-occlusive disease of the liver (Moderate) | Renal and urinary disorders | CTCAE (3.0) | Systematic Assessment |
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| Skin GVHD | Skin and subcutaneous tissue disorders | CTCAE (3.0) | Systematic Assessment |
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| Gastrointestinal GVHD | Gastrointestinal disorders | CTCAE (3.0) | Systematic Assessment |
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| Acute GVHD | General disorders | CTCAE (3.0) | Systematic Assessment | Liver, skin (rash), mucosa, and gastrointestinal tract. |
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A decision to terminate this trial was made due to the high incidence of veno-occlusive disease of the liver in participants receiving this graft-versus-host disease (GVHD) prophylaxis regimen.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Amin Alousi, MD /Professor, Stem Cell Transplantation | University of Texas (UT) MD Anderson Cancer Center | 713-745-8613 | aalousi@mdanderson.org |
| ID | Term |
|---|---|
| D019337 | Hematologic Neoplasms |
| D009190 | Myelodysplastic Syndromes |
| D007938 | Leukemia |
| D008223 | Lymphoma |
| D006086 | Graft vs Host Disease |
| ID | Term |
|---|---|
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D001855 | Bone Marrow Diseases |
| D009370 | Neoplasms by Histologic Type |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D009173 | Mycophenolic Acid |
| C512542 | thymoglobulin |
| D000961 | Antilymphocyte Serum |
| D020123 | Sirolimus |
| D033581 | Stem Cell Transplantation |
| D018380 | Hematopoietic Stem Cell Transplantation |
| D014180 | Transplantation |
| ID | Term |
|---|---|
| D002208 | Caproates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D005227 | Fatty Acids |
| D008055 | Lipids |
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| D018942 | Macrolides |
| D007783 | Lactones |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D013514 | Surgical Procedures, Operative |
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