Rett syndrome

This early-stage trial is testing the drug vorinostat in 15 girls and young women with Rett syndrome, a rare genetic disorder that affects brain development. Each participant will receive placebo and two different doses of vorinostat, acting as their own control. The study aims t…

This pilot study tests whether an antioxidant cocktail called Rett-T can improve motor function in children and adolescents with Rett syndrome, a rare genetic disorder that affects movement and coordination. Participants, aged 2 to 21 and at least partially able to walk, will rec…

This study tests a new drug called ION440 in 48 people with MECP2 duplication syndrome, a rare genetic condition that causes intellectual disability and seizures. The drug is given via a spinal injection to see if it is safe and how the body processes it. Some participants will r…

This phase II trial is testing whether mirtazapine, an antidepressant, can improve mood, sleep, and motor symptoms in people with Rett syndrome. The study will enroll 54 females aged 5 to 40 with a confirmed MECP2 mutation. Participants will receive increasing doses of mirtazapin…

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 …

This study tests an oral medicine called bionetide in 210 girls and women with Rett syndrome, a rare genetic disorder. The goal is to see if bionetide improves behavior and communication compared to a placebo over 12 weeks. Participants must be 5 to 20 years old and have a confir…

This study creates a large registry of people with Rett syndrome, a rare genetic disorder that causes severe intellectual and physical disabilities. Researchers will collect information from doctors and caregivers to understand how the disease progresses over time. The goal is to…

This study tests whether eye-tracking tasks can detect changes in attention and learning in people with Rett syndrome who are starting a new medication called trofinetide. Researchers will compare eye movements and heart rate before and after 4 weeks of treatment in 20 participan…

This observational study uses a noninvasive EEG test to measure how the brains of people with Rett syndrome respond to simple sounds. Researchers will also ask parents or caregivers about symptoms and daily functioning. The goal is to better understand brain activity patterns in …

This registry collects information from caregivers of people with Rett syndrome worldwide. Participants can track symptoms and treatments online, and researchers can use the de-identified data to better understand the condition and plan future clinical trials. The goal is to spee…

This study looks for brain activity patterns (biomarkers) in girls with Rett syndrome. Researchers will use EEG to measure brain waves and see if these patterns change with symptoms or stay stable over time. About 200 girls with Rett syndrome and typically developing girls will t…