Active clinical trials for "Scleroderma, Systemic"

Exploratory evaluation of pharmacokinetics and safety of KHK4827 in subjects with systemic sclerosis

This phase II trial studies how well giving cyclophosphamide and anti-thymocyte globulin together followed by peripheral blood stem cell transplant (PBSCT) and mycophenolate mofetil works in treating patients with systemic scleroderma (SSc). Stem cells are collected from the patient's blood and stored prior to treatment. To store the stem cells patients are given colony-stimulating factors, such as filgrastim (G-CSF) or chemotherapy (cyclophosphamide) to help stem cells move from the bone marrow to the blood so they can be collected and stored. After storage, patients are then given high-dose chemotherapy, cyclophosphamide, and immunosuppression with anti-thymocyte globulin to suppress the immune system to prepare for the transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy and immunosuppression. After the stem cells have "engrafted" and have matured enough to support the immune system at approximately 2-3 months, patients are given a medication called mycophenolate mofetil (MMF) or Myfortic. This medication is given to prevent worsening or reactivation of SSc and is referred to as maintenance therapy.

To evaluate the efficacy and safety of KHK4827 in patients with systemic sclerosis who have moderate to severe skin thickening

This study intends to carry out a prospective, multi-center cohort study based on MRI to explore the incidence of structural and functional damage of central, brain and kidney in patiant with SSc and its clinical relevance, and to search for the characteristics of serological markers of structural and functional damage of heart, brain and kidney.

Digital Ulcers (DEs), are painful open sores on the fingers and toes and are due to limited perfusion of blood vessels in patients with Scleroderma. In particular, ulcers are caused by narrowing of the arteries, resulting in reduced blood supply to the fingers, causing pain and difficult to heal leaving deep scars. DEs may be present on the rails or fingertips, on the extensor surfaces of the joints, and depending on the underlying calcification. The etiology of ulcers is multifactorial. Raynaud's ischemia, sclerosis, dry skin, calcification and local trauma can all contribute to the onset of Digital Ulcers. Spontaneous fissures or ruptures can also develop into ulcers in patients with scleroderma and severe Raynaud's disease. Whatever the reason for their appearance, DEs negatively affect the quality of life of patients as they complicate even simple daily activities, while they can lead to serious complications such as osteomyelitis or other serious soft tissue infections, up to amputation. Over the last decade, in several randomized clinical trials, DEs have been studied in detail, which are defined or classified differently in each case. The clinical treatment of patients with DE would be facilitated by the availability of specific criteria for the classification of DE. Internationally, the classification of pressure ulcers is usually followed, but this classification is not sufficient for all types of DE observed in Scleroderma. The lack of a clear classification of DEs prompted researchers to evaluate the frequency and morphology of lesions, their characteristics, their physical course, and their healing time in groups of patients with Scleroderma. In a recent study, three categories of classification of digital ulcers based on the patient's clinical picture were proposed by the UK Scleroderma Study Group (UKSSG).

Systemic sclerosis (SSc) is a heterogeneous systemic autoimmune disease with distinct prognosis according to patients. In patients with systemic sclerosis, interstitial lung disease (ILD) concerns almost 50 % of patients and represents the main cause of mortality. Janus kinases (JAK) inhibitors are recent therapies in the field of systemic autoimmune diseases, already approved in patients with rheumatoid arthritis. Use of JAK inhibitors in systemic sclerosis is based on their anti-inflammatory and anti-fibrotic properties. Several preclinical murine models of systemic sclerosis demonstrated the efficacy of ruxolitinib and tofacitinib on cutaneous and pulmonary fibrosis. Recently, tofacitinib was evaluated in SSc patients in two clinical studies and showed significant improvement on skin fibrosis. The objective of this study is to evaluate efficacy and safety of JAK inhibitors in SSc patients with ILD.

The purpose of this study is to assess feasibility, safety and preliminary efficacy of Extracorporeal Photopheresis in the treatment of active diffuse cutaneous systemic sclerosis (dcSSc). This pilot study will help to determine if further study (a RCT) is justified.

The purpose of this study is to evaluate the safety, tolerability and efficacy of iguratimod in adult subjects with diffuse cutaneous systemic sclerosis.

Systemic sclerosis and inflammatory myopathies, which sometimes combine (scleromyositis), have shared pathophysiological elements. In both diseases, many cell subtypes are involved in damage to organs such as T lymphocytes, B lymphocytes, and unconventional (non-B, non-T) lymphocytes called innate lymphoid cell (ILC). The increasing complexity of our understanding of the immune system (multiplication of recognized cell subtypes) also makes the strategies for analyzing pathophysiological mechanisms more complex. Currently, no biomarker perfectly predicts the phenotype and evolution of patients. Multi-OMIC analyzes will be performed (identification of cell populations as well as genomic, transcriptomic and proteomic characterization) in blood and tissue samples (skin and muscle biopsy) in patients with systemic sclerosis and inflammatory myopathies, with the objective of identifying discriminating molecular signatures (biomarkers) according to the characteristics of the disease and its evolution.

This is a randomized, multi-site, adaptive, open-label clinical trial comparing the immune response to different additional doses of COVID-19 vaccine in participants with autoimmune disease requiring IS medications. All study participants will have negative serologic or suboptimal responses (defined as a Roche Elecsys® Anti-SARS-CoV-2 S result ≤200 U/mL) or a low immune response (defined as a Roche Elecsys® Anti-SARS-CoV-2 S result >200 U/ml and ≤2500 U/mL) to their previous doses of COVID-19 vaccine. The study will focus on 5 autoimmune diseases in adults: Systemic Lupus Erythematosus (SLE) Rheumatoid Arthritis (RA) Multiple Sclerosis (MS) Systemic Sclerosis (SSc), and Pemphigus. This study will focus on 4 autoimmune diseases in pediatric participants: Systemic Lupus Erythematosus (SLE) Juvenile Idiopathic Arthritis (JIA) Pediatric-Onset Multiple Sclerosis (POMS) Juvenile Dermatomyositis (JDM)