Active clinical trials for "Scleroderma, Localized"

Scleroderma, or systemic sclerosis, is a chronic connective tissue disease generally classified as one of the autoimmune rheumatic diseases. The disease is characterized by thickening and fibrosis skin, affecting vessels and many organs such as the esophagus, stomach, bowls, lung, heart and kidney. The exact cause or causes of scleroderma are still unknown, but scientists and medical investigators in a wide variety of fields are working hard to make those determinations. It is known that scleroderma involves overproduction of collagen. FLICKMAN et al, in 1973 published an article about the role of lidocaine at prolyl-hydroxylase activity decrease, which is an important enzyme of collagen production. Until now, there is only a case series showing the improvement of thickening skin (75%) and esophagus symptoms (66%) after intravenous lidocaine 2% during 10 days. So it is necessary a RCT to prove these findings.

Digital ulcers (DUs) are an expression of the microangiopathy in patients with scleroderma (SSc). DUs lead to pain and impaired hand use. DUs remain a severe complication for many patients and effective therapy remains elusive. In the present study, the investigators propose to evaluate the efficacy of Sildenafil in DUs healing in a randomized double blind control study in SSc patients.

This study will evaluate the safety and efficacy of the combination of rituximab and methotrexate to treat disabling fibrosing skin disorders.Rituximab will be administered at baseline and month 6. The drug will be considered efficacious if the skin thickness diminishes substantially.

This study will evaluate the effect of treprostinil diethanolamine (UT-15C) sustained release tablets(compared to placebo) on digital ulcers in patients with scleroderma. Treprostinil diethanolamine is an analog of prostacyclin. Prostacyclin is a naturally occuring substance produced by the cells of blood vessels that inhibits platelet aggregation, induces vasodilation, and suppresses smooth muscle proliferation. Improvement in blood flow in lower limbs and fingers would be anticipated to result in a reduction in ischemic pain, Raynaud's phenomenon and promote healing of digital ulcers and other ischemic wounds.

Systemic Sclerosis (Scleroderma) varies greatly in clinical manifestations, mode of presentation, and course. The natural history of this chronic autoimmune disease ranges from benign to fatal. Patients are classified into limited and diffuse scleroderma defined by the degree of skin involvement. Patients with limited disease (e.g. the C.R.E.S.T. syndrome) generally have mild disease and normal survival. However, patients with diffuse cutaneous scleroderma often have severe multi-system disease that is not only devastating emotionally and physically but is associated with a 60-70% five year survival and a 40-50% 10 year survival. No therapies have proven effective in the treatment of scleroderma. Strategy to treat scleroderma have included attempts to prevent fibrosis with drugs that interfere with collagen metabolism, attempts to modify the disease process by immunosuppression and attempts to alter the disease by vasoactive drugs. High dose of corticosteroids and other immunosuppressive drugs (e.g. chlorambucil, 5-fluorouracil, methotrexate, cyclophosphamide, cyclosporine) used at conventional doses have not proven curative, but have shown some benefit for inflammatory features of the disease (e.g. arthritis, myositis, fibrosing alveolitis). Both allogeneic and autologous bone marrow transplantation (BMT) have shown to modify and in some instances reverse a variety of animal models of autoimmune disease. This has prompted many investigators to propose the use of peripheral blood stem cell transplantation (PBSCT) for the treatment of autoimmune disease including scleroderma. Unfortunately, this approach risks infusing untreated autoreactive lymphocyte clones after the immunoablative preparative regimen. We have previously demonstrated that high-dose cyclophosphamide without BMT can induce durable and complete remissions in another autoimmune disease, severe aplastic anemia. Recent data with high dose cyclophosphamide show that it can induce complete remissions in other autoimmune hematologic disorders. The objective of this study is to determine whether high dose cyclophosphamide can induce a durable remission in scleroderma patients with life-threatening disease, and to determine toxicity of high dose cyclophosphamide in high risk scleroderma patients.

Hand involvement in scleroderma leads to functional disability due to the relationship between grip strength, wrist and finger movement. The vast majority of patients report that their activities are restricted and their quality of life decreases for this reason. Literature indicate that more work is needed to continue to develop and evaluate rehabilitation interventions in this population. This study is a randomized controlled study examining the effects of 8 weeks of upper extremity home exercises on grip strength, normal joint movement, activity performance and functionality in patients with scleroderma. In our study, it is aimed to contribute to the standardization of upper extremity exercise protocols for scleroderma patients, to increase the quality of life of patients and to increase their independence in daily living activities.

The main ailm of this phase I-II study is to evaluate toxicity and efficacy of allogenic mesenchymal stem cell therapy to treat severe systemic sclerosis. In practice this treatment will be given to patients with a rapidly evolutive disease or refractory to cyclophosphamide.

Scleroderma,also known as systemic sclerosis (SSc), is a multisystem disease affecting skin and other tissues including joints, muscles, lungs, the gastrointestinal tract and kidneys and tissue fibrosis is widespread. SSc presents special problems for developing therapies due to the heterogeneous clinical presentation, the variability of disease progression and the difficulty quantifying the extent of disease. For most disease manifestations, treatment is primarily symptomatic and generally inadequate. This study will utilize a 4-gene biomarker of skin disease as the primary efficacy outcome in a short duration, placebo-controlled clinical trial of rilonacept, designed to provide preliminary data for a larger trial. These gene biomarkers should provide a strong surrogate for such trials in the future and, if IL-1 is indeed the cytokine leading to fibrosis in this disease, provide a highly significant start to finding a therapeutic for SSc that for the first time might dramatically affect fibrosis. A central hypothesis of this study is that IL-1 inhibition will downregulate the 4-gene biomarker over a relatively short period of time, much shorter than is historically thought necessary to see changes in the MRSS, a skin score measurement tool. Entry criteria will include the recent onset of diffuse cutaneous SSc as this is the population most likely to show progressive skin disease and also the population examined in previous studies showing correlations between MRSS and the 4-gene biomarker. Secondary outcomes will include other validated measures of SSc disease activity. MRSS and SSc health assessment questionnaire (SHAQ), will be followed during the trial. This study will also test the effect of rilonacept on global skin gene expression using microarray analyses of skin biopsies. In addition, serum biomarkers of SSc disease activity (COMP, THS-1 and IFI44) and a biomarker of inflammasome activation (CRP) will be tested before and after treatment.

Digital ulcers are one of the most prevalent complications of scleroderma (systemic sclerosis). There can be found few surveys on effect of topical agents on healing process of the ulcers. Thus, the aim of this study is to assess and compare the effects of topical diltiazem on SSc digital ulcers versus topical nitroglycerin.

A Phase II multi-center, double-blind, parallel group, randomized and placebo-controlled clinical trial addressing the treatment of patients with active and symptomatic Scleroderma-related interstitial lung disease (SSc-ILD).