Hope for rett syndrome: new drug trial targets behavior and sleep

NCT ID NCT07503444

First seen Apr 02, 2026 · Last updated May 08, 2026 · Updated 6 times

Summary

This study tests whether fenfluramine hydrochloride can improve symptoms of Rett syndrome, a rare genetic disorder that affects mostly girls. About 200 participants will receive either the drug or a placebo for 14 weeks, with an option to continue on the drug afterward. The main goals are to see if the drug reduces behavioral issues and improves overall condition, as rated by caregivers and doctors.

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Contacts and locations

Study contacts

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Conditions

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