Tofacitinib for hard-to-treat Blau syndrome
Part of Bones, joints & muscles, Immune system & allergy, Skin clinical trials.
This study tests if the drug tofacitinib (a JAK inhibitor) can help people with Blau syndrome whose symptoms did not improve with other treatments. Blau syndrome is a rare genetic condition that causes inflammation in the skin, joints, eyes, and other organs.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed NOD2 gene mutation.
- You have symptoms like skin rash, arthritis, eye inflammation, or lung involvement.
- You have tried at least one immunosuppressant or biologic for 12 weeks or more without success.
- You are not currently taking an IL-1 inhibitor or other biologic drugs.
- You are not pregnant or breastfeeding.
- You do not have severe organ failure or a life expectancy under 6 months.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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