Lifesaving drug now available for kids with rare gut disease
NCT ID NCT06003881
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This program gives patients with CHAPLE disease, a rare and life-threatening immune disorder that causes protein loss from the gut, access to the experimental drug pozelimab. The goal is to provide treatment and monitor long-term safety. Participants must have a confirmed genetic diagnosis of CD55-deficient protein-losing enteropathy.
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Conditions
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