Active clinical trials for "Scleroderma, Diffuse"

The DUAL-1 study is designed as a multicenter, double-blind two-period study with an initial fixed 16-week Period 1, followed by a Period 2 of variable duration. All patients completing Period 1 will continue on their original randomized treatment into Period 2, until the last randomized patient has completed Period 1. Patients will be randomized in a 1:1:1 ratio (macitentan 3mg: macitentan 10mg: placebo). The primary objective is to demonstrate the effect of macitentan on the reduction of the number of new digital ulcers in patients with systemic sclerosis and ongoing digital ulcers. Other objectives include: the evaluation of the efficacy of macitentan on hand functionality and DU burden at Week 16 in SSc patients with ongoing DU disease. the evaluation of the safety and tolerability of macitentan in these patients. the evaluation of the efficacy of macitentan on time to first DU complication during the entire treatment period.

Systemic sclerosis (SSc, scleroderma) is a multisystem autoimmune rheumatic disease that causes inflammation, vascular damage and fibrosis. Besides involvement of skin, fibrosis also affects lung and heart. Although advances in understanding in pathophysiology and use of immunosuppressive therapy has brought significant improvement in outcome of other autoimmune diseases, scleroderma still remains as a disease with high mortality and 10 yr survival rate has improved only from 54% to 66% during last 25 years1. The frequency of deaths due to renal crisis significantly decreased (mainly due to effectiveness of ACE Inhibitors), from 42% to 6% of scleroderma-related deaths (p 0.001), whereas the proportion of patients with scleroderma who died of pulmonary fibrosis increased (due to lack of significant treatment) from 6% to 33% (p 0.001). However, presently, trials with immunosuppressive drugs including cyclophosphamide and other targeted molecules like Bosentan and Imatinib mesylate have shown very modest results at the best and given the risk of toxicity. The investigators have conducted three clinical trials with PDE5 inhibitor Tadalafil in the refractory Raynaud's phenomenon (RP) in SSc over last 3 years and had found good response in RP, healing of digital ulcers, prevention of new digital ulcers and also observed improvement in skin tightening, endothelial dysfunction and improvement of quality of life. The investigators therefore hypothesize that tadalafil may have an efficacy in improving the ILD of SSc. The investigators therefore design this double-blind, randomized, placebo-controlled trial of oral Tadalafil (20 mg alternate day) in patients with SSc having ILD. Patients will be randomly assigned in a 1:1 ratio to receive either Tadalafil or matched placebo and will be followed up for 6 months. Prednisolone (if required for indications other than ILD) will be allowed up to 10 mg/d in all patients. Patient/s requiring more than 10 mg/d of prednisolone or equivalent dose of steroid will be excluded from the study. Patients who will fail on therapy during the study will be excluded from the study and will be asked to choose any therapeutic option from the rescue protocol. Patients with FVC ≤ 70% predicted or DLCO ≤ 70 % of predicted, Evidence of ILD on HRCT will be enrolled. The primary objective of the study will be the change in FVC (expressed as a percentage of the predicted value) from baseline values at the end of 6-months of treatment. The secondary objectives will be improvement in dyspnea, improvement in 6 min walk distance, change in DLCO, change in total lung capacity, change in the disability index of the Health Assessment Questionnaire (S HAQ), and change quality of life (SF-36), levels of NT pro-BNP and fibrosis markers.

The investigators purposes are to define the prevalence of omeprazole resistance gastroesophageal reflux disease (GERD) in systemic sclerosis (SSc), to compare the efficacy of omeprazole in combination with algycon versus omeprazole in combination with domperidone on the severity of reflux symptoms in omeprazole resistant GERD in SSc, and to compare the efficacy of omeprazole in combination with algycon versus omeprazole in combination with domperidone on the frequency of symptoms in omeprazole- resistant GERD in SSc.

PSSc-001 (LOTUSS) This study is a Phase 2, multinational, open-label, randomized, parallel-group, safety and tolerability study of pirfenidone in participants with systemic sclerosis-related interstitial lung disease (SSc-ILD).

OBJECTIVES: I. Evaluate the safety and efficacy of oral iloprost, a prostacyclin analog, in patients with Raynaud's phenomenon secondary to systemic sclerosis.

The primary objective of this study is to evaluate the safety and tolerability of intravenously administered MEDI-551 over escalating single doses in adult subjects with Scleroderma

This study investigates the efficacy and safety of STI571 for the treatment of fibrosis in participants with systemic sclerosis. Other purposes of the study were to investigate whether STI571 is effective in improving lung functions and other test results called biomarkers. Whether STI571 is well-absorbed in systemic sclerosis participants' gut was also investigated by testing the drug level in the blood (pharmacokinetics).

Purpose of the study is to evaluate the effectiveness and safety of a new investigational dur, IC351. Study is designed to gather information regarding the possible usefulness of IC351 as a treatment of several blood vessel features of scleroderma. This includes Raynaud phenomenon as well as the vaginal dryness and discomfort associated with scleroderma

Systemic Sclerosis is a disease that may be caused by the immune system reacting against skin and certain organs. It is possible, that by changing the immune system we can modify the progression of this disease. Stem cells are created in the bone marrow. They mature into different types of blood cells that are needed including red blood cells, white blood cells, and platelets. In this study, we will stimulate the bone marrow to make extra stem cells. Next we will collect the stem cells, select specific cells, and store them. We will then give high dose chemotherapy that will destroy the patients immune system. We will then give back the selected stem cells we collected. We believe that these selected stem cells may be able to "re-create" the immune system without the portion that causes Systemic Sclerosis. The purpose of this study is to try to discover if stem cell transplantation can help patients with Systemic Sclerosis. We will also try to learn what the side effects are of this treatment in patients with Systemic Sclerosis. We hope that this treatment will help to relieve the symptoms patients are experiencing, although we do not know if it will.

To evaluate the safety and tolerability of a multiple-dosed drug (MEDI-546) in adults with scleroderma.