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Sponsored by: |
National Institute of Allergy and Infectious Diseases (NIAID) |
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Information provided by: | National Institute of Allergy and Infectious Diseases (NIAID) |
ClinicalTrials.gov Identifier: | NCT00114530 |
Scleroderma, also known as systemic sclerosis (SSc), is a disabling disease. The body attacks its own tissues, causing an overproduction of collagen. Collagen is a fiber-like protein which holds all of the structures in the body together. Patients often suffer fatigue, joint swelling and/or pain, and a loss of appetite and weight.
Scleroderma can be difficult to diagnose because it overlaps with or resembles other conditions. Severe forms of the disease typically cause significant thickening and stiffening of the skin. Internal organs are affected, resulting in damage to the heart, lungs, kidneys, and gastrointestinal tract.
Severe forms of the disease, particularly rapidly progressive diffuse SSc with involvement of internal organs, are associated with a high death rate. Studies have shown poor prognosis of patients with internal organ involvement and suggest that clinicians can identify a subpopulation of SSc subjects of high risk for death who may benefit from a more aggressive therapeutic approach. The progression of SSc and the development of new organ disease despite conventional treatment is another signal for clinicians to be more aggressive in therapy.
Many treatments have been tried for severe SSc, but none has been proven effective in preventing disease progression or reversing fibrosis in randomized, controlled trials. Although the exact cause of scleroderma is unknown, the immune system is thought to play a major role in the development of the disease, and there is some evidence to suggest that immune-based therapies are beneficial. Oral cyclophosphamide taken daily had been compared to placebo in a randomized clinical trial of patients with lung disease from scleroderma. The cyclophosphamide was shown to be beneficial though the effect was small. Cyclophosphamide may improve the skin disease as well. The effectiveness of intravenous cyclophosphamide used at the doses in this study have not been evaluated in a formal controlled clinical trial.
In response to the absence of an effective treatment for SSc, autologous hematopoietic stem cell transplantation (HSCT) has been proposed as a potential therapy. Hematopoietic stem cells are immature blood cells that can develop into any of the different blood and immune cells your body uses. Researchers believe that resetting the immune system may stop the progression of the disease. The main purpose of this study is to compare the two ways of treating SSc: 1) high-dose immunosuppressive therapy (HDIT) followed by HSCT and 2) high-dose pulse IV cyclophosphamide (CTX).
Condition | Intervention | Phase |
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Scleroderma, Systemic Sclerosis Autoimmune Disease |
Procedure: Autologous stem cell transplantation and High-dose immunosuppressive therapy (HDIT) Drug: Cyclosphosphamide Drug: Equine antithymocyte globulin Drug: Methylprednisolone Drug: Growth colony stimulating factor (G-CSF) Radiation: Total body irradiation (TBI) |
Phase II Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Open Label, Active Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | Scleroderma: Cyclophosphamide or Transplantation (SCOT) |
Estimated Enrollment: | 226 |
Study Start Date: | June 2005 |
Estimated Study Completion Date: | June 2012 |
Estimated Primary Completion Date: | June 2012 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Active Comparator
This group of study participants will have hematopoietic stem cell transplantation. Please see the detailed description for more information about this group.
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Procedure: Autologous stem cell transplantation and High-dose immunosuppressive therapy (HDIT)
transplantation of blood stem cells from participant's bone marrow or blood and immunosuppressive therapy
Drug: Cyclosphosphamide
immunosuppressant, usually used in the treatment of cancer
Drug: Equine antithymocyte globulin
immunosuppressant
Drug: Methylprednisolone
corticosteroid
Drug: Growth colony stimulating factor (G-CSF)
used to increase white blood cells
Radiation: Total body irradiation (TBI)
used to eradicate the immune system thought to be causing the systemic sclerosis
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2: Active Comparator
This group of study participants will receive high doses of intravenous cyclophosphamide. Please see the detailed description for more information about this group.
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Drug: Cyclosphosphamide
immunosuppressant, usually used in the treatment of cancer
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Ages Eligible for Study: | 18 Years to 69 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Keith Sullivan, MD | 866-909-SCOT | sulli025@mc.duke.edu |
Study Chair: | Keith Sullivan, MD | Division of Cellular Therapy, Duke University |
Study Chair: | Daniel Furst, MD | Rheumatology Division, UCLA Medical School |
Study Chair: | Peter McSweeney, MD | Blood and Marrow Transplant Program, Presbyterian/St. Luke's Medical Center, Rocky Mountain Cancer Center |
Principal Investigator: | Leslie Crofford, MD | University of Kentucky |
Principal Investigator: | Maureen Mayes, MD, MPH | The University of Texas Health Science Center, Houston |
Principal Investigator: | Richard Nash, MD | Fred Hutchinson Cancer Research Center |
Responsible Party: | DAIT.NIAID ( Associate Director, Clinical Research Program ) |
Study ID Numbers: | DAIT SCSSc-01, SCOT |
Study First Received: | June 15, 2005 |
Last Updated: | August 25, 2009 |
ClinicalTrials.gov Identifier: | NCT00114530 History of Changes |
Health Authority: | United States: Food and Drug Administration |
autoimmune disease |
Anti-Inflammatory Agents Immunologic Factors Methylprednisolone Hormone Antagonists Hormones, Hormone Substitutes, and Hormone Antagonists Antiemetics Prednisolone acetate Cyclophosphamide Neuroprotective Agents Hormones Connective Tissue Diseases Scleroderma Alkylating Agents Methylprednisolone Hemisuccinate |
Autoimmune Diseases Antineoplastic Agents, Hormonal Skin Diseases Methylprednisolone acetate Sclerosis Immunosuppressive Agents Glucocorticoids Antilymphocyte Serum Prednisolone Scleroderma, Systemic Antineoplastic Agents, Alkylating Peripheral Nervous System Agents Antirheumatic Agents |
Anti-Inflammatory Agents Immunologic Factors Molecular Mechanisms of Pharmacological Action Methylprednisolone Antineoplastic Agents Physiological Effects of Drugs Hormones, Hormone Substitutes, and Hormone Antagonists Antiemetics Prednisolone acetate Cyclophosphamide Hormones Neuroprotective Agents Pathologic Processes Therapeutic Uses Connective Tissue Diseases |
Alkylating Agents Methylprednisolone Hemisuccinate Autoimmune Diseases Antineoplastic Agents, Hormonal Skin Diseases Immune System Diseases Gastrointestinal Agents Methylprednisolone acetate Sclerosis Glucocorticoids Protective Agents Immunosuppressive Agents Pharmacologic Actions Antilymphocyte Serum Autonomic Agents |