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

NCT ID NCT07218575

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests whether everolimus, a drug already approved for other conditions, can safely improve social abilities in people with PTEN Hamartoma Tumor Syndrome (PHTS). About 60 adults and children with PHTS and social difficulties will take either everolimus or a placebo for 6 months, then all will receive everolimus for another 6 months. The goal is to see if the drug helps with social skills and functioning.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

everolimus (Afinitor, Zortress)

What this could lead to

If it works, this could point toward a treatment for social difficulties in people with PTEN Hamartoma Tumor Syndrome.

What could go wrong

This is a small, early-phase trial with only 60 participants. The drug may not improve social abilities, and side effects from everolimus are possible.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

autism Bannayan-Riley-Ruvalcaba syndrome Cowden disease Lhermitte-Duclos disease PTEN hamartoma tumor syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Stanford University

    Stanford, California, 94305, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••