Hematological MalignanciesPhase 2NCT00506948
Thymoglobulin, Sirolimus and Mycophenolate Mofetil for Prevention of Acute Graft-Versus-Host Disease (GVHD)
A Phase 2 study testing Mycophenolate Mofetil (MMF), Thymoglobulin and Sirolimus in people with Hematological Malignancies and Myelodysplastic Syndrome, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Hematological Malignancies, Myelodysplastic Syndrome, Leukemia
- What is being tested?
- Mycophenolate Mofetil (MMF), Thymoglobulin and Sirolimus
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2006to Oct 2010
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- HLA-identical sibling or matched unrelated donor transplants not eligible for protocols of higher priority.
- Age 18-75 years.
- Bilirubin </=1.5 mg/dl, serum glutamic-pyruvic transaminase (SGPT) </= 200 IU/ml.
- Creatinine </=1.6 mg/dl.
Who cannot join
- Regimens including rituximab or alemtuzumab in the preparative regimen.
- Patients can not have received prior treatment with gemtuzumab.
- Planned conditioning chemotherapy for transplant can not include gemtuzumab.
- Planned conditioning chemotherapy for transplant can not include the busulfan and cyclophosphamide regimen.
- HIV seropositivity
- Uncontrolled infection, not responding to adequate antimicrobial therapy after 7 days of treatment. The protocol PI is the final arbiter of eligibility.
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- Pregnancy
- Inability to sign consent.
- Patients who are past recipients of allogeneic or autologous stem cell transplants from any source.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Thymoglobulin, Sirolimus and Mycophenolate Mofetil for Prevention of Acute GVHD Following Allogeneic Hematopoietic Stem Cell Transplantation
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.
Read the 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.
- Being tested
- Mycophenolate Mofetil (MMF), Thymoglobulin, Sirolimus, Stem Cell Transplant
- Keywords
- Hematological Malignancies, Myelodysplastic Syndrome, Graft Versus Host Disease, GVHD, Allogeneic Hematopoietic Stem Cell Transplantation, AHSCT, Leukemia, Lymphoma, T-Cells, Thymoglobulin, ATG, rATG, Rabbit Antithymocyte Globulin, Rapamune, Sirolimus, Mycophenolate Mofetil, Cellcept, MMF
- Medical subject headings
- Hematologic Neoplasms, Myelodysplastic Syndromes, Leukemia, Lymphoma, Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mycophenolate Mofetil (MMF)Drug
Also called CellCept, MMF
15 mg/kg by vein or by mouth every 12 hours.
ThymoglobulinDrug
Also called Antithymocyte Globulin, rATG, ATG
1.5 mg/kg by vein daily for 4 days
SirolimusDrug
Also called Rapamune
6 mg daily by mouth for 1 day, followed by 2 mg daily for 1 day.
Stem Cell TransplantProcedure or surgery
Also called hematopoietic stem cell transplantation, HSCT, transplantation
Stem cell infusion on Day 0.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Mycophenolate Mofetil (MMF); Drug: Thymoglobulin; Drug: Sirolimus; Procedure: Stem Cell Transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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).
Measured: Baseline to 100 days post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jul 2007 (estimated)
- Study startSep 2006
- Primary completionOct 2010
- Study completionOct 2010
Why it stopped: Halted due to high incidence of veno-oclusive disease of the liver.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hematological MalignanciesStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Responsible party
- Sponsor
- Investigators
- Amin Alousi, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00506948
- Sponsor's protocol ID
- 2006-0435
- Status last verified
- Sep 2020
- First submitted
- 20 Jul 2007
- Last updated
- 24 Sep 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Thymoglobulin, Sirolimus and Mycophenolate Mofetil for Prevention of Acute GVHD Following Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT00506948
- Acronym / sponsor ID
- 2006-0435
- Keywords
- Hematological Malignancies, Myelodysplastic Syndrome, Graft Versus Host Disease, GVHD, Allogeneic Hematopoietic Stem Cell Transplantation, AHSCT, Leukemia, Lymphoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Halted due to high incidence of veno-oclusive disease of the liver.
- Enrolment
- 13 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Study start
- Sep 2006
- Primary completion
- Oct 2010 (actual)
- Study completion
- Oct 2010 (actual)
- First posted
- 25 Jul 2007 (estimated)
- Results
- Posted 20 Feb 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Thymoglobulin + Sirolimus + MMF | Experimental | Drug: Mycophenolate Mofetil (MMF), Drug: Thymoglobulin, Drug: Sirolimus, Procedure: Stem Cell TransplantThymoglobulin 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. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
- HLA-identical sibling or matched unrelated donor transplants not eligible for protocols of higher priority.
- Age 18-75 years.
- Bilirubin </=1.5 mg/dl, serum glutamic-pyruvic transaminase (SGPT) </= 200 IU/ml.
- Creatinine </=1.6 mg/dl.
Key exclusion criteria (9 listed)
- Regimens including rituximab or alemtuzumab in the preparative regimen.
- Patients can not have received prior treatment with gemtuzumab.
- Planned conditioning chemotherapy for transplant can not include gemtuzumab.
- Planned conditioning chemotherapy for transplant can not include the busulfan and cyclophosphamide regimen.
- HIV seropositivity
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mycophenolate Mofetil (MMF)Also: CellCept, MMF15 mg/kg by vein or by mouth every 12 hours. | Drug | Thymoglobulin + Sirolimus + MMF |
| ThymoglobulinAlso: Antithymocyte Globulin, rATG, ATG1.5 mg/kg by vein daily for 4 days | Drug | Thymoglobulin + Sirolimus + MMF |
| SirolimusAlso: Rapamune6 mg daily by mouth for 1 day, followed by 2 mg daily for 1 day. | Drug | Thymoglobulin + Sirolimus + MMF |
| Stem Cell TransplantAlso: hematopoietic stem cell transplantation, HSCT, transplantationStem cell infusion on Day 0. | Procedure or surgery | Thymoglobulin + Sirolimus + MMF |
Primary outcome
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).
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).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- University of Texas (UT) MD Anderson Cancer Center Official WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Amin Alousi, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Mycophenolate Mofetil (MMF), Thymoglobulin and Sirolimus in people with Hematological Malignancies and Myelodysplastic Syndrome, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 24 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Feb 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Failure Rate (time frame: Baseline to 100 days post transplant). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2020.