Scleroderma is a rare autoimmune disease where the immune system triggers excessive collagen production, hardening the skin and sometimes internal organs. About 300,000 people in the US have it. Treatment focuses on managing symptoms and slowing organ damage, with immunosuppressants like mycophenolate and cyclophosphamide for lung and skin involvement.
What's actually going on in research
Trials are testing antifibrotic drugs that block collagen buildup, drugs that target specific immune pathways driving scleroderma, stem cell transplant for severe disease, and treatments for pulmonary arterial hypertension. Researchers are also studying whether drugs approved for lung scarring in other diseases can help scleroderma lung disease.
Antifibrotic therapies
Drugs like nintedanib, already approved for idiopathic pulmonary fibrosis, are being tested in scleroderma lung disease. Early results suggest they may slow the scarring that makes breathing difficult.
Targeted immune therapies
Newer drugs that block specific immune signals, including IL-6 inhibitors and drugs targeting B cells, are showing promise in early trials. These may work better than older immunosuppressants with fewer side effects.
Stem cell transplant
Autologous stem cell transplant, where a patient's immune system is reset with their own stem cells, has shown long-term benefits in severe scleroderma. Trials are refining which patients benefit most and how to make the procedure safer.
What to know before you search
Eligibility typically depends on disease duration, which organs are affected, extent of skin or lung involvement, and whether standard treatments have been tried.
What types of trials are currently open
- Antifibrotic trials — Testing drugs that block collagen production or break down existing scarring in the lungs or skin.
- Immunosuppression trials — Comparing different drugs that calm the overactive immune response, including newer targeted therapies versus standard immunosuppressants.
- Pulmonary hypertension trials — Testing treatments for high blood pressure in the lungs, a serious complication of scleroderma.
- Stem cell trials — Studies of stem cell transplant for people with rapidly progressing disease who haven't responded to other treatments.
- Vascular trials — Testing drugs for Raynaud's phenomenon and digital ulcers, where blood flow problems cause painful fingertip sores.
Recently added Scleroderma trials
Share your health data about pelvic floor and lung involvement
Systemic sclerosis (SSc) is a heterogeneous multisystem disease characterized by vascular dysfunction, immune abnormalities, and progressive fibrosis of the skin and internal organs. Pulmonary involvement, particularly interstitial lung disease (ILD), is one of the most common and clinically important manifestations of SSc and may lead to impaired pulmonary function, respiratory muscle weakness, reduced diaphragmatic function, and decreased exercise capacity. In addition to pulmonary involvement, systemic inflammation, physical inactivity, nutritional disturbances, and musculoskeletal manifestations may contribute to generalized skeletal muscle dysfunction in individuals with SSc. Pelvic floor dysfunction, including urinary and fecal incontinence, pelvic organ prolapse, pelvic pain, and sexual dysfunction, has also been reported in individuals with SSc. Previous studies have suggested that vascular dysfunction, tissue fibrosis, and structural and functional abnormalities of the pelvic floor may contribute to these symptoms. Furthermore, impaired pelvic floor and sexual function in women with SSc have been associated with several disease-related characteristics, including dyspnea, interstitial lung disease, reduced physical activity, functional disability, and impaired quality of life. The pelvic floor muscles form the inferior component of the core muscle system and function synergistically with the diaphragm and abdominal muscles to regulate intra-abdominal pressure. Coordinated activity between the diaphragm and pelvic floor muscles during respiration has been demonstrated. Therefore, pulmonary and respiratory muscle involvement in SSc may potentially be associated with impaired pelvic floor function. However, the relationships between pelvic floor dysfunction and pulmonary function, respiratory muscle strength, exercise capacity, and core endurance in patients with SSc with pulmonary involvement remain insufficiently understood. Investigating these relationships may contribute to a more comprehensive understanding of pelvic floor dysfunction and its associated factors in this population.
Take an experimental immune therapy for autoimmune disease
The primary objectives of this trial are to evaluate the safety and tolerability and to determine the maximum tolerated dose (MTD) or recommended Phase 2 dose of FT839 with or without rituximab and/or background therapy and/or conditioning therapy.
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