Lupus Immunosuppressive/Immunomodulatory Therapy or Stem Cell Transplant (LIST)
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|ClinicalTrials.gov Identifier: NCT00230035|
Recruitment Status : Withdrawn (Recommended by DSMB due to lack of accrual)
First Posted : September 30, 2005
Last Update Posted : February 1, 2013
Systemic lupus erythematosus, also known as lupus or SLE, is a chronic, multisystem, autoimmune disease in which the body's internal system of defense attacks its own normal tissues. This abnormal autoimmune response can result in damage to many parts of the body, especially the skin, joints, lungs, heart, brain, intestines, and kidneys. Both genetic and environmental risk factors are involved in the development of lupus, but these are poorly understood.
SLE has an overall 10-year survival between 80 and 90%. However, we estimate that severe lupus not responding to the usual available treatments has a 50% mortality rate in 10 years. Kidney problems occur in 30% to 50% of lupus patients and may progress to kidney failure. Kidney disease due to lupus occurs more frequently in African-Americans and Hispanics. Lupus can affect many parts of the body and cause damage, but the severe form can result in death from kidney disease; cardiovascular disease, specifically atherosclerosis; central nervous system disease; and infections.
Currently, no single standard therapy for treatment of severe SLE exists. Usually physicians prescribe an aggressive regimen of one or a combination of immunosuppressive/immunomodulatory treatments. This approach to therapy for all forms of severe SLE derives largely from studies of lupus nephritis. Current treatment, although effective in many people, are not effective in all patients and are associated with drug-induced morbidity. The design of the control arm for this study reflects the current status of treatment of SLE in the academic setting. Investigators may choose from a list of commonly used and currently available immunosuppressive/immunomodulatory treatments to optimize the treatment of their patients, based on their past treatment history and response to those treatments. Study treatments may consist of corticosteroids, cyclophosphamide (CTX), azathioprine, methotrexate, cyclosporine, mycophenolate mofetil (MMF), plasmapheresis, intravenous immunoglobulin (IVIG), rituximab, and leflunomide. Treatment may be changed as frequently and as necessary within the first year of the study to control the manifestations of SLE in each patient. New therapies that become available during the course of this trial may be added to the list of approved medications for this study.
In response to the absence of a uniformly effective treatment for severe lupus, autologous hematopoietic stem cell transplantation (HSCT) has been proposed as a potential therapy. Hematopoietic stem cells are immature blood cells that can develop into all of the different blood and immune cells the body uses. Researchers believe that resetting the immune system may stop or slow down the progression of the disease. The main purpose of this study is to compare two ways of treating SLE: 1) high-dose immunosuppressive therapy (HDIT) followed by HSCT and 2) currently available immunosuppressive/immunomodulatory therapies.
|Condition or disease||Intervention/treatment||Phase|
|Systemic Lupus Erythematosus||Procedure: Leukapheresis Procedure: Non-myeloablative high dose immunosuppressive therapy conditioning (HDIT) Procedure: Autologous CD34+HPC transplantation (HSCT) Procedure: Plasmapheresis Drug: Rabbit anti-thymocyte globulin Drug: Methylprednisolone Drug: Growth colony stimulating factor (G-CSF) Drug: Corticosteroids Drug: Mycophenolate mofetil Drug: Azathioprine Drug: Intravenous immunoglobulin Drug: Methotrexate Drug: Rituximab Drug: Leflunomide||Phase 2|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Randomized, Open Label, Phase II Multicenter Study of Non-Myeloablative Autologous Transplantation With Auto-CD34+HPC Versus Currently Available Immunosuppressive/Immunomodulatory Therapy for Treatment of Systemic Lupus Erythematosus|
|Study Start Date :||September 2005|
high dose immunosuppressie therapy (HDIT) followed by HSCT (hemopoietic stem cell transplantation).
|Procedure: Leukapheresis Procedure: Non-myeloablative high dose immunosuppressive therapy conditioning (HDIT) Procedure: Autologous CD34+HPC transplantation (HSCT)|
Active Comparator: 2
Currently available immunosuppressive/immunomodulatory therapy
|Procedure: Plasmapheresis Drug: Rabbit anti-thymocyte globulin Drug: Methylprednisolone Drug: Growth colony stimulating factor (G-CSF) Drug: Corticosteroids Drug: Mycophenolate mofetil Drug: Azathioprine Drug: Intravenous immunoglobulin Drug: Methotrexate Drug: Rituximab Drug: Leflunomide|
- Mortality resulting from treatment, underlying disease, or unrelated causes [ Time Frame: At Month 30 ]
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 ClinicalTrials.gov identifier (NCT number): NCT00230035
|United States, California|
|UCSD, Thornton Hospital|
|La Jolla, California, United States, 92037-0943|
|UCLA, Rehabilitation Center|
|Los Angeles, California, United States, 90095-1670|
|United States, Illinois|
|Chicago, Illinois, United States, 60611|
|United States, New York|
|Feinstein Institute for Medical Research NS-LIJ Health System|
|Manhassat, New York, United States, 11030|
|United States, North Carolina|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27709|
|Study Chair:||Richard Burt, MD||Division of Immunotherapy, Northwestern University|
|Study Chair:||Bevra Hahn, MD||Division of Rheumatology, Department of Medicine, University of California, Los Angeles|
|Study Chair:||Kenneth Kalunian, MD||Division of Rheumatology, Allergy, and Immunology, University of California, Los Angeles|
|Study Chair:||Ann Traynor, MD||Division of Hematology and Oncology, University of Massachusetts Medical School|
|Study Chair:||Keith Sullivan, MD||Division of Cellular Therapy, Department of Medicine, Duke University|
|Study Chair:||Betty Diamond, MD||Department of Medicine, Columbia University|