Fenfluramine HCl for Rett Syndrome in a Phase 3 study

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3 trial evaluates the efficacy of fenfluramine hydrochloride versus placebo in participants with Rett syndrome. Eligible individuals have a confirmed MECP2 mutation, stable functional status for at least 6 months, and a Rett Syndrome Clinical Severity Scale range of 10–36 with a CGIS of 4 or higher. The study uses a randomized, controlled design to determine if the drug can improve core symptoms or functioning. Locations include sites in Arkansas and Florida under lead sponsor UCB Biopharma.

Why it matters

Who can join: the full rules

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

Where it's running

• Ep0247 21010
  Little Rock, Arkansas, United States Recruiting
• Ep0247 21009
  Miami, Florida, United States Recruiting
• Ep0247 21011
  Orlando, Florida, United States Recruiting
• Ep0247 21007
  Atlanta, Georgia, United States Recruiting
• Ep0247 21003
  Philadelphia, Pennsylvania, United States Recruiting
• Ep0247 21005
  Houston, Texas, United States Recruiting

Study contacts

• Study coordinator: UCB Cares
• Email: ucbcares@ucb.com
• Phone: +18445992273

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Auditory EEG and behavioral testing for people with Rett syndrome

  This project will record noninvasive EEG while people with Rett syndrome and matched controls listen to simple sounds to see how their brains process sound.

  Recruiting University of Rochester · Rochester, New York · NCT07569445

• Daily oral vorinostat for girls and young women with Rett syndrome

  This trial will try daily oral vorinostat at two escalating dose levels, using a four-week placebo baseline and an N-of-1 design so each participant serves as her own control, to see if the drug is safe and shows clinical or molecular benefit in girls and young women with typical Rett syndrome.

  Recruiting Phase 1 Unravel Biosciences, Inc. · Medellín · NCT07150013

• Efficacy of Bionetide for treating Rett syndrome in females

  This study is testing if an oral medication called Bionetide can help improve behavior and communication in girls and young women aged 5 to 20 with Rett syndrome.

  Recruiting at 18 sites Phase 3 Biomed Industries, Inc. · Phoenix, Arizona and 17 other locations · NCT06840496

• Assessing cognitive function in Rett syndrome patients receiving trofinetide treatment

  This study is testing how well eye-tracking tasks can measure cognitive abilities in people with Rett syndrome who are starting treatment with trofinetide.

  Recruiting University of Minnesota · Minneapolis, Minnesota · NCT06705816

• Identifying biomarkers in Rett Syndrome

  This study is trying to find specific brain signals in girls with Rett Syndrome to see how they differ from girls without the condition and how these signals relate to the severity of their symptoms over time.

  Recruiting at 6 sites Children's Hospital of Philadelphia · Los Angeles, California and 5 other locations · NCT05932589

• Gene editing to correct common MECP2 mutations in Rett syndrome

  This project will test CRISPR/Cas9 delivered with AAV to correct common MECP2 mutations in females with Rett syndrome using lab and animal work.

  Recruiting University of Siena · Siena, Siena · NCT05740761

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.