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Could a cancer drug boost social abilities in rare genetic disorder?

NCT ID NCT07218575

First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 24 times

Summary

This study tests whether the drug everolimus can safely improve social abilities in adults and children with PTEN Hamartoma Tumor Syndrome (PHTS), a genetic condition that raises cancer risk and often causes autism-like social difficulties. Half of the 60 participants will receive everolimus for 6 months, while the other half gets a placebo; then everyone gets the drug for another 6 months. The study measures social skills through questionnaires and checkups over one year.

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