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Tracking Information | |||||||||
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First Received Date † | February 7, 2007 | ||||||||
Last Updated Date | February 7, 2007 | ||||||||
Start Date † | December 2006 | ||||||||
Current Primary Outcome Measures † |
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Original Primary Outcome Measures † | Same as current | ||||||||
Change History | No Changes Posted | ||||||||
Current Secondary Outcome Measures † |
Secondary evaluations of response. Scleroderma Health Assessment Questionnaire (SHAQ): The SHAQ will be evaluated at study entry compared to 6 and 12 months. | ||||||||
Original Secondary Outcome Measures † | Same as current | ||||||||
Descriptive Information | |||||||||
Brief Title † | Mycophenolate Mofetil in Systemic Sclerosis | ||||||||
Official Title † | Phase I, Open-Label Study of Mycophenolate Mofetil In Systemic Sclerosis | ||||||||
Brief Summary | 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. |
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Detailed Description | 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. |
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Study Phase | Phase I | ||||||||
Study Type † | Interventional | ||||||||
Study Design † | Treatment, Non-Randomized, Open Label, Active Control, Single Group Assignment, Safety/Efficacy Study | ||||||||
Condition † | Diffuse Cutaneous Systemic Sclerosis | ||||||||
Intervention † | Drug: Mycophenolate Mofetil | ||||||||
Study Arms / Comparison Groups | |||||||||
Publications * | |||||||||
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline. |
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Recruitment Information | |||||||||
Recruitment Status † | Recruiting | ||||||||
Enrollment † | 30 | ||||||||
Completion Date | |||||||||
Primary Completion Date | |||||||||
Eligibility Criteria † | Inclusion Criteria:
SKIN SUBSTUDY (20 patients)
OR, MUSCLE SUBSTUDY (10 patients)
Exclusion Criteria:
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Gender | Both | ||||||||
Ages | 18 Years and older | ||||||||
Accepts Healthy Volunteers | No | ||||||||
Contacts †† |
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Location Countries † | United States | ||||||||
Expanded Access Status | |||||||||
Administrative Information | |||||||||
NCT ID † | NCT00433186 | ||||||||
Responsible Party | |||||||||
Secondary IDs †† | |||||||||
Study Sponsor † | Boston University | ||||||||
Collaborators †† | Aspreva Pharmaceuticals | ||||||||
Investigators † |
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Information Provided By | Boston University | ||||||||
Verification Date | February 2007 | ||||||||
† Required WHO trial registration data element. †† WHO trial registration data element that is required only if it exists. |