A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
Recruiting
Phase 3Interventional Study
Rett Syndrome
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
5 – 35
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
200 (estimated)
Sponsor
UCB BIOSCIENCES, Inc. · Industry
Who this trial is looking for
This trial is looking for people with Rett syndrome to see if fenfluramine can help. Participants will be monitored and complete caregiver assessments during the study.
Are You a Good Fit for This Trial?
You may be able to join if
I have been diagnosed with typical Rett Syndrome.
I have a mutation in the MECP2 gene.
I haven't lost any skills in walking, hand function, or communication for at least 6 months.
I have a Rett Syndrome Clinical Severity Scale rating between 10 and 36.
I have a Clinical Global Impression-Severity score of 4 or higher.
I am between 5 and 35 years old.
I have a caregiver who can help me during the study.
You may not be able to join if
I have had any type of cancer in the last 5 years, except some skin cancers.
I have major issues with my vital signs.
I have significant heart problems that don't allow me to join the study.
I have a serious medical condition like chronic lung disease.
I need more than 4 ant seizure medications.
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
Answer three quick questions written from this study's own rules to see if you may qualify. No account needed to find out.
The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).
Trial Locations
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Status
Eligibility Criteria
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 postregress…
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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