Clinical Trials Logo

Clinical Trial 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.


Clinical Trial 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. ;


Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


NCT number NCT00433186
Study type Interventional
Source Boston University
Contact
Status Completed
Phase Phase 1
Start date March 2006
Completion date March 2011

See also
  Status Clinical Trial Phase
Completed NCT02161406 - A Study of Subcutaneous Abatacept to Treat Diffuse Cutaneous Systemic Sclerosis Phase 2
Completed NCT03222492 - Brentuximab Vedotin for Systemic Sclerosis Phase 1/Phase 2
Completed NCT04137224 - Safety and Pharmacokinetics of IgPro20 and IgPro10 in Adults With Systemic Sclerosis (SSc) Phase 2
Not yet recruiting NCT04986605 - Extracorporeal Photopheresis in Early Diffuse Cutaneous Systemic Sclerosis Phase 2
Completed NCT02503644 - Proof-of-concept Trial of IVA337 in Diffuse Cutaneous Systemic Sclerosis Phase 2
Recruiting NCT04781543 - A Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients With Diffuse Cutaneous Systemic Sclerosis Phase 2
Enrolling by invitation NCT05626751 - An Open-label Extension Trial of HZNP-HZN-825-301 in Adult Participants With Diffuse Cutaneous Systemic Sclerosis (Diffuse Cutaneous SSc) Phase 2
Withdrawn NCT05098145 - A Safety and Efficacy Study of FCR001 in Adults With Rapidly Progressive Diffuse Cutaneous Systemic Sclerosis Phase 1/Phase 2
Terminated NCT04478994 - A Study With TEPEZZA in Patients With Diffuse Cutaneous Systemic Sclerosis (dcSSc) Phase 1
Completed NCT03198689 - Brentuximab Vedotin in Early Diffuse Cutaneous Systemic Sclerosis Phase 2
Terminated NCT04680975 - Efficacy and Safety of Belumosudil in Subjects With Diffuse Cutaneous Systemic Sclerosis Phase 2
Terminated NCT03919799 - KD025 in Subjects With Diffuse Cutaneous Systemic Sclerosis Phase 2
Not yet recruiting NCT06152172 - CARTIMMUNE: Study of Patients With Autoimmune Diseases Receiving KYV-101 Phase 1
Suspended NCT04166552 - Evaluation of Safety, Tolerability and Preliminary Efficacy of EHP-101 in Diffuse Cutaneous Systemic Sclerosis Phase 2
Not yet recruiting NCT06375005 - Efficacy and Safety of Telitacicept in the Treatment of Systemic Sclerosis Phase 2
Recruiting NCT05270668 - Phase 2 Safety and Efficacy Study of Tulisokibart (MK-7240/PRA023) in Subjects With Systemic Sclerosis Associated With Interstitial Lung Disease (SSc-ILD) (MK-7240-007) Phase 2
Recruiting NCT05168215 - Effectiveness of Bosentan Therapy in Patients With Systemic scleRosis and Progressive Digital ulcEr Disease.
Completed NCT04440592 - Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Diffuse Cutaneous Systemic Sclerosis Phase 2
Recruiting NCT05149768 - Open Label Extension Study of Brentuximab Vedotin in Early dcSSc Phase 2
Withdrawn NCT04138485 - Efficacy and Safety of IgPro10 in Adults With Systemic Sclerosis (SSc) Phase 2