New Treatments & Clinical Trials for Scleroderma

Scleroderma encompasses both limited and diffuse systemic sclerosis as well as localized scleroderma, all involving abnormal collagen deposition that stiffens and thickens skin and — in systemic forms — internal organs. The most feared complications are pulmonary fibrosis and pulmonary arterial hypertension.

What's actually going on in research

The field is moving toward earlier and more aggressive treatment in patients with diffuse disease identified by high-risk autoantibodies. Trials are testing combination approaches pairing antifibrotic drugs with immunosuppressives rather than single-agent therapy. B-cell depletion with rituximab and newer anti-BAFF drugs are being tested across skin and lung manifestations. Stem cell transplantation for carefully selected severe early-diffuse disease continues to show long-term benefit in follow-up studies.

What to know before you search

Eligibility requires confirmed systemic sclerosis, subtype classification (limited vs. diffuse), specific autoantibodies, disease duration, and organ involvement assessment.

What types of trials are currently open

• Immunosuppression trials — Testing mycophenolate, cyclophosphamide, rituximab, and tocilizumab for skin and organ fibrosis.
• Anti-fibrotic trials — Evaluating nintedanib, pirfenidone, and novel TGF-beta pathway blockers.
• Combination therapy trials — Testing paired immunosuppressive and antifibrotic approaches for greater disease control.
• Lung transplant trials — Evaluating outcomes and patient selection for lung transplantation in end-stage scleroderma lung disease.
• Vascular trials — Testing treatments for Raynaud's phenomenon, digital ulcers, and pulmonary arterial hypertension.