Ignite Creation Date:
2024-05-05 @ 5:20 PM
Last Modification Date:
2024-10-26 @ 9:30 AM
Study NCT ID:
NCT00433186
Status:
COMPLETED
Last Update Posted:
2011-03-31
First Post:
2007-02-07
Brief Title:
Mycophenolate Mofetil in Systemic Sclerosis
Sponsor:
Boston University
Organization:
Boston University
Study Overview
Official Title:
Phase I Open-label Study of Mycophenolate Mofetil In Systemic Sclerosis
Status:
COMPLETED
Status Verified Date:
2011-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
Detailed Description:
Systemic sclerosis SSc is an autoimmuneconnective 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None