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A study to learn if fenfluramine hydrochloride is safe and works in people with Rett syndrome
EU CTIS ID: 2025-523157-34-00
What this study is testing
To investigate the efficacy of fenfluramine HCl versus placebo in study participants with Rett syndrome (RTT)
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Participant has typical or classic Rett Syndrome (RTT) according to the RettSearch Consortium 2010 revised criteria
- Participant has a documented disease-causing mutation 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.
You likely can't join if
- 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: a. Greater than trace aortic valve regurgitation. b. Greater than mild mitral valve regurgitation. c. Possible signs of pulmonary arterial hypertension (PAH) with abnormal pulmonary artery systolic pressure (PASP) or PASP ≥35 mmHg. d. Evidence of left ventricular dysfunction (systolic or diastolic). e. 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
See the full eligibility criteria
Who can join
- Participant has typical or classic Rett Syndrome (RTT) according to the RettSearch Consortium 2010 revised criteria
- Participant has a documented disease-causing mutation 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.
What rules you out
- 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: a. Greater than trace aortic valve regurgitation. b. Greater than mild mitral valve regurgitation. c. Possible signs of pulmonary arterial hypertension (PAH) with abnormal pulmonary artery systolic pressure (PASP) or PASP ≥35 mmHg. d. Evidence of left ventricular dysfunction (systolic or diastolic). e. 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
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Japan
- United Arab Emirates
- United Kingdom
- Brazil
- China
- Colombia
- Australia
- Mexico
- Korea, Republic of
- Saudi Arabia
- Chile
- Canada
- Israel
- Georgia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Japan; United Arab Emirates; United Kingdom; Brazil; China and 9 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.