Can a Weight-Loss drug ease rett syndrome?

NCT ID NCT07503444

Not yet recruiting Symptom relief Sponsor: UCB BIOSCIENCES, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 10, 2026 · Updated 1 time

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Rett syndrome

As listed by the trial registrant

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

RETT SYNDROME

Contacts and locations

Locations

Ep0247 11105

Madrid, Spain
Ep0247 15001

Budapest, Hungary
Ep0247 15002

Debrecen, Hungary
Ep0247 19002

Krakow, Poland
Ep0247 21005

Houston, Texas, 77030, United States
Ep0247 21010

Little Rock, Arkansas, 72202, United States
Ep0247 21011

Orlando, Florida, 32806, United States
Ep0247 21013

Irving, Texas, 75062, United States
Ep0247 43003

Setagaya-ku, Japan
Ep0247 43006

Musashimurayama-shi, Japan

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