Can a Weight-Loss drug ease rett syndrome?
NCT ID NCT07503444
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This Phase 3 trial tests whether fenfluramine (Fintepla) can reduce behavioral and emotional symptoms in people with Rett syndrome. About 200 participants will receive either the drug or a placebo for 14 weeks. The study measures changes in behavior, sleep, and overall condition using caregiver questionnaires and clinician ratings.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
fenfluramine hydrochloride (also known as Fintepla)
What this could lead to
If successful, this could provide a new treatment option to ease behavioral and emotional symptoms in Rett syndrome.
What could go wrong
This is an early-stage Phase 3 trial with only 200 participants. Results may not show significant benefit over placebo, and fenfluramine has known risks including heart valve issues and pulmonary hypertension.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ep0247 11105
Madrid, Spain
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Ep0247 15001
Budapest, Hungary
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Ep0247 15002
Debrecen, Hungary
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Ep0247 19002
Krakow, Poland
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Ep0247 21005
Houston, Texas, 77030, United States
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Ep0247 21010
Little Rock, Arkansas, 72202, United States
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Ep0247 21011
Orlando, Florida, 32806, United States
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Ep0247 43003
Setagaya-ku, Japan
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Ep0247 43006
Musashimurayama-shi, Japan