View clinical trials related to Scleroderma, Diffuse.
Filter by:Systemic sclerosis (SSc) is a multisystem connective tissue disease characterised by vascular abnormalities and fibrosis, including those of the skin and can be categorised as either Limited cutaneous scleroderma or Diffuse cutaneous scleroderma. It is estimated that more than 90% of patients with SSc experience Raynaud's phenomenon (RP) at regular intervals during the course of their disease. Approximately 50% of patients with SSc develop severe digital ischaemia and/or ulceration which seems to be painful, difficult to heal, susceptible to infections and heavily influences quality of life and increases SSc-related disability. Medical treatment is commonly used as an effective first line approach in the NHS policy when uncontrolled RP attacks emerge. However, considering the short-term side effects (oedema, headaches, heart palpitations, dizziness and constipation) but also the long-term side effects of nifedipine (heart dysfunction and increased cardiovascular risk) as well as the financial cost of this approach, alternative approaches with less side effects and less cost implications are warranted. An alternative approach would be to implement a programme of therapeutic exercise that would be suitable for this patient group. To the investigators knowledge the efficacy of exercise on microcirculation in RP has not been previously examined. In this regard, high intensity interval training (HIIT) has come to prominence over the last years for its effectiveness in inducing greater improvements in vascular function than moderate intensity continuous training. Due to the variation in HIIT protocols evidence is limited to support which protocol is the most effective in SSc patients. Moreover, it should be noted that the chief aim of the research project is to encourage long-term adherence to physical activity and rehabilitation programmes in these patients which might be beneficial for the vascular function. A short HIIT protocol (30seconds/passive recovery) may elicit more favourable patient reported satisfaction /enjoyment levels compared to other longer exercise duration protocols. A short HIIT protocol (30seconds/passive recovery) has demonstrated to be well tolerated, preferred protocol with a low perception of effort, patient comfort and with a longer time spent at high percentage of V̇O2peak than a longer HIIT protocol with active recovery phases in chronic heart failure patients. More recent evidence supports this notion; when enjoyment levels in an overweight/obese cohort were examined after a short HIIT protocol. Although it is known that HIIT is capable to improve vascular function and potentially the microcirculatory parameters, evidence is scarce regarding the mode of exercise that will be more effective on digital microcirculation where the RP attacks are present in SSc patients. Assumptions could be made that utilising an upper-body exercise would potentially be more beneficial for the digital microcirculation rather than lower-body exercise where the working muscles promote the blood flow in the lower limbs. Hence, the differential effects that may occur by the upper- and lower-limb exercise on the digital microcirculation in SSc patients should be examined. Resistance training (RT) alone has shown significant improvements in the function of the vasculature; moreover, a combination of aerobic and RT have shown both in the past and recently important enhances in the vascular function and microcirculation. However, the limited number of studies have investigated the effects of RT on vasculature bespeaks a lack of concrete evidence. Moreover, to the investigators knowledge the effects of combined exercise (RT and aerobic exercise) utilising a HIIT protocol and RT on microcirculation has yet to be examined. Aims: The primary aim of the present study is to examine the feasibility of exercise in patients with Systemic Sclerosis experiencing Raynaud's Phenomenon.
The study will be carried out on 50 consecutive consenting patients of systemic sclerosis with PAH recruited from outpatient department of internal medicine and rheumatology clinic of PGIMER, Chandigarh, India It is a single centre double blind randomised controlled trial evaluating the effect of upfront dual therapy (sildenafil and bosentan) vs monotherapy (sildenafil) Participants will be randomised in 1:1 ratio to one of treatment arms. Placebo and PDE-5 inhibitors 20 mg BD to 60 mg if patient tolerates the drug well to one study arm and PDE-5 inhibitors plus ER antagonist 62.5 to max of 125 to other study group
GSK2330811 is a humanized monoclonal antibody which is in development for systemic sclerosis (SSc), a rare autoimmune disease with high morbidity and mortality. Currently, there are no approved disease modifying therapies and it is an area of high unmet medical need. GSK2330811 has been shown to bind and neutralize Oncostatin M (OSM) that has been associated with fibrosis, vasculopathy and inflammation in a number of diseases. This multi-center, randomized, double-blind (sponsor open), placebo controlled, proof of mechanism study will be the first study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of repeat subcutaneous (SC) doses of GSK2330811 in male and female participants with diffuse cutaneous SSc (dcSSc). Participants with active disease and a disease duration of <= 60 months will be enrolled. Approximately 24 to 40 participants will be randomized across two sequential cohorts. Cohort 1 will evaluate a repeat-dose predicted to provide sub-maximal inhibition of OSM, leading to a dose escalation decision. Cohort 1 is planned to consist of at least 4 participants, randomized such that 3 participants will receive GSK2330811 100 milligram (mg) and 1 will receive placebo. Cohort 2 is planned to consist of at least 20 participants, randomized such that participants will receive GSK2330811 300 mg and placebo in a 3:1 ratio respectively. The duration of the study is up to 34 weeks including a screening period of up to 6 weeks, treatment period of 12 weeks and follow-up period of 16 weeks.
Objective: To compare the efficacy of topical 10% nifedipine versus 5% sildenafil in patients with secondary Raynaud's phenomenon (RP). Methods: A randomized, double-blind, placebo-controlled pilot study took place in 10 patients with secondary RP. Topical 10% nifedipine on one hand and 5% sildenafil on the other hand were applied. The thumbs didn't receive any cream and served as a control group. The primary outcome was the improvement of blood flow and vessel diameter of the digital arteries measured by high frequency color Doppler ultrasound before and 1 hour after treatment.
Efficacy and safety of local infusion of botulinum toxin type B in patients with systemic sclerosis (SSc) with digital ulcer is evaluated by a randomized, double-blind study.
A double-blinded, placebo-controlled study of Dimethyl fumarate (DMF) in 34 Systemic Sclerosis-Pulmonary Hypertension (SSc-PAH) patients. The study will determine safety and the primary outcome variability for DMF in treating SSc-PAH; the primary outcome of clinical efficacy in this pilot trial will be improvement in 6-minute walk distance (6MWD).
Systemic sclerosis (SSc) is a rare autoimmune disease, mainly characterized by cutaneous and visceral fibrosis. Digital ulcer and sclerosing skin are commonly affected on hands, but the treatment for these manifestations are often ineffective. Adipose tissue contains stromal vascular fraction (SVF), which is abundant multipotent stem cells, capable of tissue repair. A prior study (NCT01813279) has shown the safety and tolerance at 6 months of the subcutaneous injection of SVF in the fingers in SSc. There are only few ways to manage SSc patients with skin lesion who already have treated with several medications (including vasodilators, PDE5 inhibitor, endothelin receptor antagonist) but some times their skin lesions are critical physically and emotionally. Autologous SVF injection could be one of the treatment options to treat skin lesion of SSc. Thus, the investigators study the efficacy and potential adverse event in Korean patients with SSc.
Primary Objective: To evaluate, in comparison with placebo, the efficacy of SAR156597 administered subcutaneously for 24 weeks on skin fibrosis in participants with diffuse cutaneous systemic sclerosis (dcSSc). Secondary Objectives: - To evaluate the efficacy of SAR156597 compared to placebo on physical/functional disability in participants with dcSSc. - To evaluate the efficacy of SAR156597 compared to placebo on respiratory function of participants with dcSSc. - To evaluate the safety profile of SAR156597 compared to placebo in participants with dcSSc. - To evaluate the potential for immunogenicity (anti-drug antibodies response) of SAR156597 in participants with dcSSc. - To evaluate the pharmacokinetics (trough plasma concentrations) of SAR156597 administered subcutaneously for 24 weeks.
The primary objective of this study is to provide preliminary data on the efficacy (digital ulcer net burden) and safety of riociguat administered 3 times daily (TID) in comparison to placebo in patients with scleroderma-associated digital ulcers
Systemic sclerosis is a multisystem disease and can involve the lungs in the form of ILD. Lung involvement is the most common cause of death in these patients. The present study is performed to study the efficacy of oral mycophenolate mofetil in treating early and mild ILD in patients of SSc. The efficacy and side effects of mycophenolate mofetil will be compared with that of oral placebo.