Rett syndrome – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:24:35 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Rett syndrome – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 A Phase 3 Study of Fenfluramine Hydrochloride for Safety and Efficacy in Patients with Rett Syndrome https://clinicaltrials.eu/trial/a-phase-3-study-of-fenfluramine-hydrochloride-for-safety-and-efficacy-in-patients-with-rett-syndrome/ Wed, 27 May 2026 04:05:20 +0000 https://clinicaltrials.eu/trial/a-phase-3-study-of-fenfluramine-hydrochloride-for-safety-and-efficacy-in-patients-with-rett-syndrome/ The study focuses on Rett syndrome, a rare neurological condition that usually appears in early childhood and leads to difficulties with movement, speech, and learning. The medication being tested is an oral liquid called fenfluramine hydrochloride, which is given by mouth at a dose based on body weight. A matching placebo with no active ingredient is also used for comparison.

The main goal of the trial is to find out whether the study drug is safe and can improve the symptoms of Rett syndrome. Participants are randomly assigned to receive either the medication or the placebo, and neither the participants nor the researchers know which one is being given. The treatment period lasts about three months, during which caregivers and clinicians complete questionnaires about behavior, sleep, and communication, and routine heart checks are performed. After the initial phase, participants may continue in an open‑label extension where all receive the active medication, allowing further observation of long‑term safety and benefit.

]]> Study on the Effects of Mirtazapine for Improving Symptoms in Patients with Rett Syndrome https://clinicaltrials.eu/trial/study-on-the-effects-of-mirtazapine-for-improving-symptoms-in-patients-with-rett-syndrome/ Wed, 29 Apr 2026 15:03:06 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-mirtazapine-for-improving-symptoms-in-patients-with-rett-syndrome/ This clinical trial is focused on studying the effects of the medication mirtazapine in individuals with Rett syndrome. Rett syndrome is a rare genetic disorder that affects brain development, leading to severe physical and mental disabilities. The study aims to see if mirtazapine can help improve social behavior, facial and breathing functions, and physical movements in those affected by this condition.

Participants in the study will receive mirtazapine, which is given as an oral solution. The study will observe how the medication affects symptoms based on the age and severity of the condition in each participant. The goal is to determine which symptoms mirtazapine can improve and to what extent.

The study will take place over a period of time, during which participants will be monitored for changes in their condition. The effectiveness of the treatment will be assessed using various scales and measurements, such as the Motor-Behavior Assessment Scale and the Anxiety, Depression, and Mood Scale. The study will also look at improvements in sleep patterns, hand function, and overall behavior. The results will help understand the potential benefits of mirtazapine for those living with Rett syndrome.

]]> Study on the Safety and Tolerability of Leriglitazone for Children with Rett Syndrome https://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-leriglitazone-for-children-with-rett-syndrome/ Wed, 29 Apr 2026 14:58:31 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-leriglitazone-for-children-with-rett-syndrome/ This clinical trial is focused on studying Rett Syndrome, a rare genetic disorder that affects brain development, leading to severe physical and cognitive impairments. The study will evaluate a treatment called leriglitazone, which is provided as an oral suspension. The purpose of the study is to assess the safety and tolerability of leriglitazone in children with Rett Syndrome.

Participants in the study will be randomly assigned to receive either leriglitazone or a placebo, which looks like the treatment but does not contain the active substance. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This approach helps ensure that the results are not biased. The study will last for a period of up to 36 weeks, during which participants will take the oral suspension daily.

Throughout the study, various assessments will be conducted to monitor the participants’ health and any changes in their condition. These assessments will include questionnaires and scales that measure behavior, motor skills, communication, and overall clinical impressions. The study will also track the number of seizures, apnea episodes, and hyperventilation episodes experienced by participants each week. The goal is to gather comprehensive data on how leriglitazone affects individuals with Rett Syndrome and to determine its potential benefits and risks.

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