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Can a Weight-Loss drug ease rett syndrome?

NCT ID NCT07503444

Symptom relief Sponsor: UCB BIOSCIENCES, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 18, 2026 · Updated 4 times

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 200 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2026

Expected to finish

Aug 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

5 to 35 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Participant has typical or classic Rett Syndrome (RTT) according to the RettSearch Consortium 2010 revised criteria * Participant has a documented disease-causing mutation or deletion in the methyl-CpG-binding protein 2 (MECP2) gene * Participant meets criteria for postregression for at least 6 months prior to Screening, defined as: * No loss or degradation of ambulation (including gait, coordination, or independence of walking/standing); * No loss or degradation of hand function; no loss or degradation of speech (including babbling, words, or previously developed communicative vocalizations); * No loss or degradation of nonverbal communicative or social skills (including eye gaze, using body to indicate communicative intent, or social attentiveness) * Participant has an Rett Syndrome Clinical Severity Scale (RTT-CSS) rating of 10 to 36 (inclusive) * Participant has a Clinical Global Impression-Severity (CGIS) score of ≥4 * Participant has a legal representative capable of providing signed informed consent on behalf of the participant as described in the protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. * Participant is aged 5 to 35 years of age (inclusive) at the time of first administration of investigational intervention. * Male or female. * Participant has a consistent caregiver who is ≥18 years of age at the Screening Visit. The caregiver needs to be able to complete the caregiver assessments defined for the entire study. Every attempt should be made to have the same evaluator complete the assessments for the duration of the study. Exclusion Criteria: * Participant has a history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years. * Participant has clinically significant abnormality in vital signs according to the Investigator * Participant has an exclusionary cardiovascular or cardiopulmonary abnormality based on echocardiogram (ECHO), electrocardiogram (ECG), or physical examination, and is not approved for entry by the central cardiac reader. Exclusionary abnormalities include, but are not limited to: 1. Greater than trace aortic valve regurgitation. 2. Greater than mild mitral valve regurgitation. 3. Possible signs of pulmonary arterial hypertension (PAH) with abnormal pulmonary artery systolic pressure (PASP) or PASP ≥35 mmHg. 4. Evidence of left ventricular dysfunction (systolic or diastolic). 5. Clinically significant structural cardiac abnormality, including but not limited to mitral valve prolapse, atrial or ventricular septal defects, or patent ductus arteriosus with reversal of shunt (right to left shunt). Note: Patent foramen ovale without a reversal of shunt or a bicuspid aortic valve is not considered exclusionary * Participant has a clinically significant medical condition, including chronic obstructive pulmonary disease, interstitial lung disease, portal hypertension, or need for invasive mechanical ventilation (eg, via tracheostomy), or has had clinically relevant symptoms or a clinically significant illness currently or in the 4 weeks prior to the Screening Visit that would negatively impact study participation, collection of study data, or pose a risk to the participant * Participant is taking \>4 concomitant antiseizure medications (ASMs). Rescue medications are not included in the count

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    23 sites in 8 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Ep0247 11001

    RECRUITING

    Brussels, Belgium

  • Ep0247 11102

    RECRUITING

    Seville, Spain

  • Ep0247 11103

    RECRUITING

    Málaga, Spain

  • Ep0247 11105

    RECRUITING

    Madrid, Spain

  • Ep0247 12005

    RECRUITING

    Brest, France

  • Ep0247 12006

    RECRUITING

    Paris, France

  • Ep0247 15001

    RECRUITING

    Budapest, Hungary

  • Ep0247 15002

    RECRUITING

    Debrecen, Hungary

  • Ep0247 17002

    RECRUITING

    Pisa, Italy

  • Ep0247 17006

    RECRUITING

    Troina, Italy

  • Ep0247 17008

    RECRUITING

    Naples, Italy

  • Ep0247 19002

    RECRUITING

    Krakow, Poland

  • Ep0247 21005

    RECRUITING

    Houston, Texas, 77030, United States

  • Ep0247 21007

    RECRUITING

    Atlanta, Georgia, 30322, United States

  • Ep0247 21009

    RECRUITING

    Miami, Florida, 33155, United States

  • Ep0247 21010

    RECRUITING

    Little Rock, Arkansas, 72202, United States

  • Ep0247 21011

    RECRUITING

    Orlando, Florida, 32806, United States

  • Ep0247 21013

    RECRUITING

    Irving, Texas, 75062, United States

  • Ep0247 43001

    RECRUITING

    Kurume, Japan

  • Ep0247 43002

    RECRUITING

    Osaka, Japan

  • Ep0247 43003

    RECRUITING

    Setagaya-ku, Japan

  • Ep0247 43004

    RECRUITING

    Kodaira, Japan

  • Ep0247 43006

    RECRUITING

    Musashimurayama-shi, Japan

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