Clinical Trials Using Sirolimus

Clinical trials are research studies that involve people. The clinical trials on this list are studying Sirolimus. All trials on the list are supported by NCI.

NCI’s basic information about clinical trials explains the types and phases of trials and how they are carried out. Clinical trials look at new ways to prevent, detect, or treat disease. You may want to think about taking part in a clinical trial. Talk to your doctor for help in deciding if one is right for you.

Trials 1-25 of 39
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  • A Study of Ruxolitinib vs Best Available Therapy (BAT) in Patients With Steroid-refractory Chronic Graft vs. Host Disease (GvHD) After Bone Marrow Transplantation (REACH3)

    The purpose of this study is to assess the efficacy of ruxolitinib against best available therapy in participants with steroid-refractory chronic graft-versus-host disease (SR cGvHD).
    Location: 23 locations

  • HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide

    This is a multi-center, single arm Phase II study of hematopoietic cell transplantation (HCT) using human leukocyte antigen (HLA)-mismatched unrelated bone marrow transplantation donors and post-transplantation cyclophosphamide (PTCy), sirolimus and mycophenolate mofetil (MMF) for graft versus host disease (GVHD) prophylaxis in patients with hematologic malignancies.
    Location: 6 locations

  • Sirolimus, Cyclosporine, and Mycophenolate Mofetil in Preventing Graft-versus-Host Disease in Treating Patients with Blood Cancer Undergoing Donor Peripheral Blood Stem Cell Transplant

    This phase II trial studies how well sirolimus, cyclosporine and mycophenolate mofetil works in preventing graft-vs-host disease (GVHD) in patients with blood cancer undergoing donor peripheral blood stem cell (PBSC) transplant. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving total-body irradiation together with sirolimus, cyclosporine, and mycophenolate mofetil before and after transplant may stop this from happening.
    Location: 3 locations

  • Pexidartinib and Sirolimus in Treating Patients with Sarcoma or Malignant Peripheral Nerve Sheath Tumors That Cannot Be Removed by Surgery

    This phase 1 / 2 trial studies the side effects and best dose of pexidartinib and sirolimus when given together and to see how well they work in treating patients with sarcoma or malignant peripheral nerve sheath tumors (MPNSTs) that has spread from the primary site (place where it started) to other places in the body (metastatic) or cannot be removed by surgery. Pexidartinib and sirolimus may stop the growth of sarcoma or MPNSTs by blocking the growth of new blood vessels necessary for tumor growth.
    Location: 3 locations

  • Sirolimus and Metronomic Chemotherapy in Treating Younger Patients with Recurrent and / or Refractory Solid or Central Nervous System Tumors

    This phase II trial studies how well sirolimus and continuous or frequent treatment with low doses of chemotherapy work in treating younger patients with solid or central nervous system (CNS) tumors that have come back or have not responded to previous treatment. Biological therapies, such as sirolimus, use substances made from living organisms that may stimulate or suppress the immune system in different ways and stop tumor cells from growing. Drugs used in chemotherapy, such as etoposide, celecoxib, and cyclophosphamide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving sirolimus together with metronomic chemotherapy may be an effective treatment for solid and CNS tumors.
    Location: 3 locations

  • Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients with Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders

    This phase I / II trial studies the side effects of fludarabine phosphate, cyclophosphamide and total-body irradiation followed by donor bone marrow transplant and cyclophosphamide, mycophenolate mofetil, tacrolimus, and sirolimus in treating patients with primary immunodeficiency disorders or noncancerous inherited disorders. Giving low doses of chemotherapy and total-body irradiation before a bone marrow transplant helps prepare the patient’s body to accept the incoming donor’s bone marrow and decrease the risk that the patient's immune system will reject the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can attack the body's normal cells called graft versus host disease. Giving cyclophosphamide, mycophenolate mofetil, tacrolimus, and sirolimus after the transplant may help decrease this from happening.
    Location: 3 locations

  • DEC-205 / NY-ESO-1 Fusion Protein CDX-1401, Poly ICLC, and Epacadostat in Treating Patients with Ovarian, Fallopian Tube, or Primary Peritoneal Cancer in Remission

    This partially randomized phase I / IIb trial studies the side effects and best dose of epacadostat in combination with DEC-205 / NY-ESO-1 fusion protein CDX-1401 and poly ICLC and to see how well they work in treating patients with ovarian, fallopian tube, or primary peritoneal cancer who no longer have evidence of disease. Antigens (such as cancer / testis antigen [NY-ESO-1] protein) are found on many cancer cells. Vaccines made from NY-ESO-1 protein may cause the immune system to produce immune cells and antibodies that may help locate the NY-ESO-1 and / or cancer / testis antigen 2 (LAGE-1) antigens on cancer cells. By finding them, the immune system may then work to control or eliminate the remaining cancer cells. Epacadostat is an inhibitor of an enzyme called indoleamine 2,3 dioxygenase (IDO). This enzyme is produced by tumor cells to disable immune cells, and limit the efficacy of immune attack. Giving DEC-205 / NY-ESO-1 fusion protein CDX-1401 with poly ICLC and epacadostat may generate stronger and more long lasting anti-cancer immune responses in patients with ovarian, fallopian tube, and primary peritoneal cancer in remission.
    Location: 2 locations

  • Sirolimus and Auranofin in Treating Patients With Advanced or Recurrent Non-Small Cell Lung Cancer or Small Cell Lung Cancer

    This phase I / II trial studies the side effects and best dose of auranofin when given together with sirolimus and to see how well it works in treating patients with lung cancer that has spread or other places in the body and cannot be cured or controlled by treatment or has come back after a period of time during which the cancer could not be detected. Auranofin and sirolimus may stop or slow the growth of lung cancer.
    Location: 2 locations

  • A Pilot Study Evaluating the Use of mTor Inhibitor Sirolimus in Children and Young Adults With Desmoid-Type Fibromatosis

    Desmoid-type fibromatosis (or desmoid tumor) represents an intermediate grade neoplasm with a striking predilection for locally invasive growth and recurrence following resection. It occurs in children as well as young adults. As a typically localized disease, the historical standard of care for treatment has been surgical resection, with or without ionizing radiation. In some cases where surgical resection or radiation is not feasible, chemotherapy has been used. Two clinical trials conducted in the Pediatric Oncology Group (POG) and the Children's Oncology Group (COG) evaluated the role for either low intensity or non-cytotoxic chemotherapy for children with desmoid tumor that is not amenable to standard therapy. These were largely empirical treatment strategies or based on somewhat anecdotal observations. By better understanding desmoid tumor biology, even more effective therapy targeting a particular protein that is central to the disease can be developed. Desmoid tumor is well-known to be associated with deregulation of the Adenomatous Polyposis Cell / beta-catenin (APC / β-catenin pathway). This is true of familial cases associated with Gardner's Syndrome and also in sporadic desmoid tumor, nearly all of which display histological or molecular evidence of Adenomatous Polyposis Cell / beta-catenin (APC β-catenin) pathway activation (Alman et al., 1997; Lips et al., 2009). Several new pieces of evidence support the concept that deregulation of the mammalian target of rapamycin (mTOR) cell proliferation / survival pathway may play an important role in tumor biology when the APC / β-catenin pathway is disrupted. Sirolimus, a drug that inhibits mammalian target of rapamycin (mTOR), is currently being evaluated as an anti-cancer agent in a variety of tumor types, but it has not been previously studied in desmoid tumor. The investigators are conducting this pilot study to begin to explore whether mTOR inhibition may be beneficial for children and young adults with desmoid tumor.
    Location: 2 locations

  • Virotherapy and Natural History Study of KHSV-Associated Multricentric Castleman s Disease With Correlates of Disease Activity

    This study will gain information about a rare disorder called KSHV-associated multicentric Castleman s disease (MCD). KSHV, a virus, causes several kinds of cancer, including some forms of MCD. KSHV stands for the Kaposi s sarcoma herpes virus, also called human herpes virus-8, or HHV-8. Researchers want to understand the biology of KSHV-MCD to identify how this disease causes illness and to find ways to treat it. There is no standard therapy effective for all cases of KSHV-MCD. The disease is often fatal, and about half the people who have it die within 2 years of diagnosis. Patients ages 12 and older may be eligible for this study. Participation entails more drawing of blood and having repeated tumor biopsies than if patients received treatment in a non-research setting. Researchers would like to learn more about the relationship of KSHV and Castleman s disease symptoms, and they want to obtain at least three biopsies in this study. There are some side effects of experimental therapy that patients may take for KSHV-MCD. Zidovudine, or Retrovir , is used at a high dose. It is given orally or through a vein, four times daily, for 7 days or longer. Zidovudine can cause nausea, vomiting, decreased bone marrow function, and decreased blood counts. Combined with valganciclovir, or Valcyte , it is likely to be more toxic to bone marrow. Valganciclovir can cause problems with bone marrow function, leading to low blood counts, sterility, and defects in a fetus. Combined with zidovudine, valganciclovir may cause more toxicity to the bone marrow. It is given twice daily for 7 days or longer. Bortezomib, or Velcade , is given for a few seconds by a rapid push through a needle into the vein. It is given twice weekly for four doses and then stopped for 1 week. Bortezomib can sometimes cause low blood pressure; it also can cause gastrointestinal problems and a low blood platelet count. Rituximab and liposomal doxorubicin are drugs given by a catheter into a vein. Interferon-alpha is given by injection into the skin. Those drugs are not experimental, but their use in Castleman s disease is experimental. Some patients may be treated with a combination of chemotherapy followed by interferon-alpha. Interferon-alpha is infected into the skin by a needle. The natural form of interferon is produced by the body and helps to control viral infections. KSHV decreases the effect of the body s interferon, and the researchers want to see if giving higher doses of interferon will help to control KSHV infection. A positron emission tomography (PET) scan, for research purposes only, may be done up to three times a year. A radioactive sugar molecule called fluorodeoxyglucose, or FDG, is used. It is believed that activated lymphocytes that may be found in patients disease might use more FDG because these cells burn more glucose fuel. Children younger than 18 years will not have PET scan done. This study may or may not have a direct benefit for participants. However, detailed assessments made throughout the study may provide information to help the doctors treat KSHV-MCD better.
    Location: 2 locations

  • Trial of eRapa in Prostate Cancer Patients

    This study is to determine the safety, pharmacokinetics / pharmacodynamics, and immunologic impact of encapsulated rapamycin in patients with low risk prostate cancer under active surveillance. There will be four groups of patients, each receiving a different dose of rapamycin.
    Location: Cancer Therapy and Research Center at The UT Health Science Center at San Antonio, San Antonio, Texas

  • Donor Stem Cell Transplant following Chemotherapy and Total Body Irradiation in Treating Participants with High Risk Non-Malignant Diseases

    This phase II trial studies the side effects of a donor stem cell transplant following chemotherapy and total body irradiation in treating participants with high risk non-malignant diseases. Giving chemotherapy and total-body irradiation before a donor transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and disease cells. It may also stop the participant's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the participant they may help the participant's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving cyclophosphamide, sirolimus, and mycophenolate mofetil after the transplant may stop this from happening. Undergoing a donor stem cell transplant following chemotherapy and total body irradiation may provide therapeutic benefit or cure for participants with high risk non-malignant diseases.
    Location: University of Minnesota / Masonic Cancer Center, Minneapolis, Minnesota

  • Comparison of Triple GVHD Prophylaxis Regimens for Nonmyeloablative or Reduced Intensity Conditioning Unrelated Mobilized Blood Cell Transplantation

    This randomized phase II trial includes a blood stem cell transplant from an unrelated donor to treat blood cancer. The treatment also includes chemotherapy drugs, but in lower doses than conventional (standard) stem cell transplants. The researchers will compare two different drug combinations used to reduce the risk of a common but serious complication called "graft versus host disease" (GVHD) following the transplant. Two drugs, cyclosporine (CSP) and sirolimus (SIR), will be combined with either mycophenolate mofetil (MMF) or post-transplant cyclophosphamide (PTCy). This part of the transplant procedure is the main research focus of the study.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • 211^At-BC8-B10 before Donor Stem Cell Transplant in Treating Patients with High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, or Myelodysplastic Syndrome

    This phase I / II trial studies the side effects and best dose of 211^astatine(At)-BC8-B10 before donor stem cell transplant in treating patients with high-risk acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndrome. Radioactive substances, such as astatine-211, linked to monoclonal antibodies, such as BC8, can bind to cancer cells and give off radiation which may help kill cancer cells and have less of an effect on healthy cells before donor stem cell transplant.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • Cyclophosphamide and Sirolimus and in Treating Patients with Metastatic, Radioiodine-Refractory Thyroid Cancer

    This phase II trial studies how well cyclophosphamide and sirolimus work in treating patients with thyroid cancer that has spread to other places in the body and does not respond to treatment with radioiodine. Drugs used in chemotherapy, such as cyclophosphamide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Sirolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving cyclophosphamide and sirolimus may work better in treating patients with thyroid cancer.
    Location: University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan

  • Ruxolitinib Phosphate and Chemotherapy Given before and after Reduced Intensity Donor Stem Cell Transplant in Treating Patients with Myelofibrosis

    This pilot clinical trial studies the side effects and best dose of ruxolitinib phosphate when given together with chemotherapy before and after a donor stem cell transplant in treating patients with myelofibrosis. Ruxolitinib phosphate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as fludarabine phosphate and melphalan, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving ruxolitinib phosphate together with chemotherapy before and after a donor stem cell transplant may help stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient’s immune cells and help destroy any remaining cancer cells.
    Location: City of Hope Comprehensive Cancer Center, Duarte, California

  • Blinatumomab and T Cell Depleted Donor Blood Cell Transplant in Treating Younger Patients with Relapsed or Refractory Hematologic Malignancy after a Previous Transplant

    This phase II trial studies how well blinatumomab and T cell depleted donor blood cell transplant work in treating children and young adults with hematologic cancer that has not responded or has come back after a previous transplant. White blood cells from donors may be able to kill cancer cells in patients with hematologic cancer. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Removing the T cells from the donor cells before the transplant may stop this from happening. Monoclonal antibodies, such as blinatumomab, may interfere with the ability of cancer cells to grow and spread. Giving blinatumomab after a blood cell transplant may destroy any remaining cancer cells.
    Location: St. Jude Children's Research Hospital, Memphis, Tennessee

  • Early Allogeneic Hematopoietic Cell Transplantation in Treating Patients with Relapsed or Refractory High-Grade Myeloid Neoplasms

    This early phase I trial studies how well early stem cell transplantation works in treating patients with high-grade myeloid neoplasms that has come back after a period of improvement or does not respond to treatment. Drugs used in chemotherapy, such as filgrastim, cladribine, cytarabine and mitoxantrone hydrochloride, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy before a donor peripheral blood cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient’s immune cells and help destroy any remaining cancer cells. Early stem cell transplantation may result in more successful treatment for patients with high-grade myeloid neoplasms.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • Cyclophosphamide, Fludarabine Phosphate, and Total-Body Irradiation with or without Anti-Thymocyte Globulin before Donor Umbilical Cord Blood Transplant in Treating Patients with Hematologic Cancer

    This phase II trial studies the side effects of cyclophosphamide, fludarabine phosphate, and total-body irradiation with or without anti-thymocyte globulin before donor umbilical cord blood transplant and to see how well they work in treating patients with hematologic cancer. Giving chemotherapy and total-body irradiation before donor umbilical cord blood transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving sirolimus and mycophenolate mofetil before and after the transplant may stop this from happening.
    Location: University of Minnesota / Masonic Cancer Center, Minneapolis, Minnesota

  • Biomarkers in Predicting Treatment Response to Sirolimus and Combination Chemotherapy in Patients with High-Risk Acute Myeloid Leukemia

    This phase II trial studies whether biomarkers (biological molecules) in bone marrow samples can predict treatment response to sirolimus and combination chemotherapy in patients with acute myeloid leukemia (AML) that is likely to come back or spread (high-risk). Sirolimus inhibits or blocks the pathway that causes cancer cells to grow. Drugs used in chemotherapy, such as mitoxantrone hydrochloride, etoposide, and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Adding sirolimus to combination chemotherapy may help improve patient response. Studying samples of bone marrow from patients treated with sirolimus in the laboratory may help doctors learn whether sirolimus reverses or turns off that pathway and whether changes in biomarker levels can predict how well patients will respond to treatment.
    Location: Thomas Jefferson University Hospital, Philadelphia, Pennsylvania

  • Pilot Trial of Allogeneic Blood or Marrow Transplanation for Primary Immunodeficiencies

    Background: Allogeneic blood or marrow transplant is when stem cells are taken from one person s blood or bone marrow and given to another person. Researchers think this may help people with immune system problems. Objective: To see if allogeneic blood or bone marrow transplant is safe and effective in treating people with primary immunodeficiencies. Eligibility: Donors: Healthy people ages 4 or older Recipients: People ages 4-75 with a primary immunodeficiency that may be treated with allogeneic blood or marrow transplant Design: Participants will be screened with medical history, physical exam, and blood tests. Participants will have urine tests, EKG, and chest x-ray. Donors will have: Bone marrow harvest: With anesthesia, marrow is taken by a needle in the hipbone. OR Blood collection: They will have several drug injections over 5-7 days. Blood is taken by IV in one arm, circulates through a machine to remove stem cells, and returned by IV in the other arm. Possible vein assessment or pre-anesthesia evaluation Recipients will have: Lung test, heart tests, radiology scans, CT scans, and dental exam Possible tissue biopsies or lumbar puncture Bone marrow and a small piece of bone removed by needle in the hipbone. Chemotherapy 1-2 weeks before transplant day Donor stem cell donation through a catheter put into a vein in the chest or neck Several-week hospital stay. They will take medications and may need blood transfusions and additional procedures. After discharge, recipients will: Remain near the clinic for about 3 months. They will have weekly visits and may require hospital readmission. Have multiple follow-up visits to the clinic in the first 6 months, and less frequently for at least 5 years.
    Location: National Institutes of Health Clinical Center, Bethesda, Maryland

  • Sirolimus, Docetaxel, and Carboplatin in Treating Patients with Metastatic Hormone-Resistant Prostate Cancer

    This partially randomized phase I / II trial studies the side effects and how well sirolimus works when given together with docetaxel and carboplatin in treating patients with hormone-resistant prostate cancer that has spread to other places in the body. Biological therapies, such as sirolimus, use substances made from living organisms that may stimulate or suppress the immune system in different ways and stop tumor cells from growing. Drugs used in chemotherapy, such as docetaxel and carboplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving sirolimus together with docetaxel and carboplatin may kill more tumor cells.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • Study of Palifermin (Kepivance) in Persons Undergoing Unrelated Donor Allogeneic Hematopoietic Cell Transplantation

    Background: - In allogeneic stem cell transplantation (SCT), stem cells are taken from a donor and given to a recipient. Sometimes the recipient s immune system destroys the donor s cells. Or donor immune cells attack the recipient s tissues, called graft-versus-host disease (GVHD). This is less likely when the recipient and donor have similar human leukocyte antigens (HLA). Researchers want to see if the drug palifermin improves the results of allogeneic SCT from HLA-matched unrelated donors. Objective: - To see if high doses of palifermin before chemotherapy are safe, prevent chronic GVHD, and improve immune function after transplant. Eligibility: - Adults 18 70 with blood or bone marrow cancer with no HLA-matched sibling, but with a possible HLA-matched donor. Design: - Participants will be screened with medical history, physical exam, and blood and urine tests. They will have scans and heart and lung exams. - Before transplant, participants will: - Have many tests and exams. These include blood tests throughout the study and bone marrow biopsy. - Get a central line catheter if they do not have one. - Have 1 3 rounds of chemotherapy. - Take more tests to make sure they can have the transplant, including medical history, physical exam, and CT scan. - Get palifermin by IV and more chemotherapy. They will get other drugs, some they will take for 6 months. - Participants will get the SCT. - After transplant, participants will: - Be hospitalized at least 3 4 weeks. - Have tests for GVHD at 60 days and 6 months. These include mouth and skin photos and biopsies. - Stay near D.C. for 3 months. - Visit NIH 5 times the first 2 years, then yearly. They may have scans and biopsies.
    Location: National Institutes of Health Clinical Center, Bethesda, Maryland

  • Nonmyeloablative Donor Stem Cell Transplant in Treating Patients with Congenital Anemias including Sickle Cell Disease and Thalassemia

    This phase II trial studies the safety and efficacy of a nonmyeloablative (bone marrow will not be completely destroyed) donor stem cell transplant in treating patients with congenital (condition or trait present at birth) anemias including sickle cell disease and thalassemia. Giving low doses of total-body irradiation before a donor stem cell transplant may help stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving immunosuppressive therapies, such as alemtuzumab and sirolimus, before transplant may stop this from happening.
    Location: UT Southwestern / Simmons Cancer Center-Dallas, Dallas, Texas

  • Azacitidine and Sirolimus in Treating Patients with High Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia That Is Relapsed or Refractory or Not Eligible for Intensive Chemotherapy

    This phase II trial studies how well azacitidine and sirolimus work in treating patients with myelodysplastic syndrome that is likely to come back or spread (high-risk) or acute myeloid leukemia that has come back (relapsed) or is not responding to treatment (refractory) or is not eligible for intensive chemotherapy. Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Sirolimus may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving azacitidine with sirolimus may kill more cancer cells.
    Location: Thomas Jefferson University Hospital, Philadelphia, Pennsylvania


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