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Sponsors and Collaborators: |
Boston University Aspreva Pharmaceuticals |
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Information provided by: | Boston University |
ClinicalTrials.gov Identifier: | NCT00433186 |
This is a research study of an investigational product called Mycophenolate mofetil (MMF). The study is designed to establish the safety and potential benefit of MMF. MMF has proven one of the most effective medications to date for SLE and associated nephritis. It also appears to be active in polymyositis and dermatomyositis. This medication inhibits inosine monophosphate dehydrogenase, the rate-limiting enzyme in synthesis of guanosine nucleotides. It blocks the type II isoform found in activated lymphocytes more potently than the type I isoform inhibiting both T- and B-lymphocytes. In SSc, MMF has been tried after anti-thymocyte globulin in one small open label study with efficacy with a significant improvement in skin score. We will test the safety and efficacy of MMF in SSc. All study patients will receive the study medication. The effect of the study medication will be examined in two subgroups of patients: those with early or progressive skin disease (skin substudy) and those with muscle disease (muscle substudy). The change in modified Rodnan skin score (MRSS) and creatinine phosphokinase (CK) for, respectively, the skin and muscle substudies at 6 months after treatment will be compared to baseline values.
Condition | Intervention | Phase |
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Diffuse Cutaneous Systemic Sclerosis |
Drug: Mycophenolate Mofetil |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Active Control, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Phase I, Open-Label Study of Mycophenolate Mofetil In Systemic Sclerosis |
Estimated Enrollment: | 30 |
Study Start Date: | December 2006 |
Systemic sclerosis (SSc) is an autoimmune/connective tissue disease with complex pathogenesis involving immune system dysregulation, leading to fibrosis. Inflammatory and autoimmune aspects of this disease overlap systemic lupus erythematosus (SLE), a disease shown clearly to respond to MMF. Activated T-cells, the probable target of MMF in SLE, likely also play an important role in SSc pathogenesis. Evidence for this includes the similarity of SSc skin disease to chronic graft versus host disease, a disease in which T-cells play a critical role. MMF has proven one of the most effective medications to date for SLE and associated nephritis [1]. It also appears to be active in polymyositis and dermatomyositis, disease that also show significant overlap with SSc [2]. Myositis can also be a feature of SSc, suggesting that his disease manifestation might be particularly likely to respond to MMF. MMF inhibits inosine monophosphate dehydrogenase, the rate-limiting enzyme in synthesis of guanosine nucleotides. It blocks the type II isoform found in activated lymphocytes more potently than the type I isoform inhibiting both T- and B-lymphocytes [3]. In SSc, mycophenolate has been tried after anti-thymocyte globulin in one small open label study with efficacy with a significant improvement in skin score [4]. However, MMF has not been tried alone inSSc and has not been tried in muscle disease associated with SSc. In this study, we will test the safety and efficacy of MMF in SSc. In this study all study patients will receive the medication.
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
SKIN SUBSTUDY (20 patients)
OR, MUSCLE SUBSTUDY (10 patients)
Have a serum creatinine phosphokinase (CK) greater than 2 times the upper limit of normal
Exclusion Criteria:
Contact: Robert Lafyatis, MD | (617) 638-4312 | |
Contact: Kerry A Viger, LPN | (617) 638-4344 | kviger@bu.edu |
United States, Massachusetts | |
Boston University School of Medicine | Recruiting |
Boston, Massachusetts, United States, 02118 | |
Contact: Robert Lafyatis, MD 617-638-4312 | |
Contact: Kerry A. Viger, LPN (617) 638-4344 kviger@bu.edu | |
Principal Investigator: Robert Lafyatis, MD | |
Sub-Investigator: Robert W Simms, MD | |
Sub-Investigator: Eugene Kissin, MD | |
Sub-Investigator: Peter A Merkel, MD, MPH | |
Sub-Investigator: Michael York, MD |
Principal Investigator: | Robert Lafyatis, MD | Boston University School od Medicine Rheumatology Section |
Study ID Numbers: | H-25973 |
Study First Received: | February 7, 2007 |
Last Updated: | February 7, 2007 |
ClinicalTrials.gov Identifier: | NCT00433186 History of Changes |
Health Authority: | United States: Institutional Review Board |
Scleroderma |
Anti-Bacterial Agents Immunologic Factors Skin Diseases Mycophenolic Acid Mycophenolate mofetil Connective Tissue Diseases |
Scleroderma Scleroderma, Diffuse Sclerosis Scleroderma, Systemic Diffuse Systemic Sclerosis Immunosuppressive Agents |
Molecular Mechanisms of Pharmacological Action Skin Diseases Immunologic Factors Antineoplastic Agents Physiological Effects of Drugs Mycophenolic Acid Enzyme Inhibitors Sclerosis Antibiotics, Antineoplastic |
Immunosuppressive Agents Pharmacologic Actions Pathologic Processes Therapeutic Uses Connective Tissue Diseases Mycophenolate mofetil Scleroderma, Diffuse Scleroderma, Systemic |