Condition

Rett Syndrome clinical trials and research

• 11Clinical trials
• 30NIH research projects
• 16Countries

Newest Rett Syndrome clinical trials

All recruiting trials (11)

• 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

• Fenfluramine HCl for Rett Syndrome in a Phase 3 study

  This study tests whether fenfluramine hydrochloride helps people with Rett syndrome when compared to a placebo.

  Recruiting at 40 sites Phase 3 UCB Pharma · Little Rock, Arkansas and 39 other locations · NCT07503444

• 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

• Registry for individuals with Rett syndrome

  This study is gathering information from people with Rett syndrome and their caregivers to help improve care and develop new treatments for the condition.

  Recruiting at 19 sites International Rett Syndrome Foundation · Birmingham, Alabama and 18 other locations · NCT05432349

• A global registry for caregivers of individuals with Rett syndrome

  This study is creating a platform for caregivers of people with Rett syndrome to share their experiences and track care strategies to help improve understanding and treatment of the condition.

  Recruiting Rett Syndrome Research Trust · Trumbull, Connecticut · NCT04900493

• Observing the health management of patients with rare genetic syndromes

  This study looks at how patients with rare genetic syndromes like Prader-Willi and Klinefelter manage their health as they move from children's care to adult care to see what support they need.

  Recruiting Erasmus Medical Center · Rotterdam, South Holland · NCT04463316

• Antioxidant Cocktail vs. Placebo for Rett Syndrome in Children and Teens

  This study tests Rett-T, an antioxidant cocktail, to see if it can help core motor problems in girls and young women with Rett syndrome.

  Recruiting Phase 2 Holland Bloorview Kids Rehabilitation Hospital · Toronto, Ontario · NCT04041713

NIH-funded research (30)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Neuronal Cholesterol Enzyme CYP46A1 and Rett Syndrome biology

  A study in Rett syndrome explores whether tweaking a brain cholesterol enzyme called CYP46A1 could help with the disease by affecting brain pathways involved in Rett.

  NIH research Syracuse University · Syracuse, United States · NIH-11465463

• Neurophysiological biomarkers to speed Rett syndrome therapies

  A study to find measurable brain signals that show whether treatments are helping children with Rett syndrome.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11416178

• MeCP2 reactivation on the inactive X to treat Rett syndrome

  This project explores turning on the silent MECP2 gene on the inactive X chromosome to help people with Rett syndrome feel better.

  NIH research Columbia University Health Sciences · New York, United States · NIH-11401010

• Glial signaling and breathing in Rett syndrome

  Aims to understand how brain support cells (glia) in Rett syndrome affect breathing and whether IGF-1 related signals can improve it.

  NIH research University of Connecticut Storrs · Storrs-Mansfield, United States · NIH-11389898

• Studying how deep brain stimulation helps Rett syndrome in mice

  Researchers are testing how deep brain stimulation affects brain cells and memory in a mouse model of Rett syndrome to uncover mechanisms that could guide future therapies.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11380840

• Molecular Pathways Behind Rett Syndrome in Humans

  A patient-focused look at how changes in the MECP2 gene affect brain function and development in Rett syndrome.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11366658

• Understanding Rett Syndrome: How MECP2 Mutations Drive Brain Disease

  This grant explores how mutations in MECP2 cause Rett syndrome and aims to uncover the brain changes that lead to intellectual disability in affected individuals.

  NIH research University of Pennsylvania · Philadelphia, United States · NIH-11364233

• Therapeutic gene editing approach for Rett syndrome in girls and young patients

  A gene-editing approach for Rett syndrome (MECP2 mutations) aims to improve symptoms in children affected by this X-linked neurodevelopmental disorder.

  NIH research Jackson Laboratory · Bar Harbor, United States · NIH-11326256

• Waking up the healthy MECP2 gene to treat Rett syndrome with an XIST-targeting ASO

  A treatment that aims to wake up the healthy MECP2 gene on the silent X chromosome to help girls with Rett syndrome.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11322750

• High-precision gene correction for MECP2-related Rett syndrome

  This project plans to use a high-precision, viral-based genome editing method to repair MECP2 gene changes in people with Rett syndrome.

  NIH research Beckman Research Institute/city of Hope · Duarte, United States · NIH-11310866

• How cerebellar support cells (Bergmann glia) may affect Rett syndrome

  Researchers will look at whether problems in specific cerebellar support cells called Bergmann glia cause the movement and social difficulties seen in people with Rett syndrome.

  NIH research Henry Ford Health + Michigan State University Health Sciences · East Lansing, United States · NIH-11251632

• Deep brain stimulation for Rett syndrome

  Researchers are testing whether deep brain stimulation can stimulate new brain cells and improve memory and movement for people with Rett syndrome.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11159649

• Correcting genetic mutations in Rett syndrome using advanced genome editing techniques

  This study is exploring a new way to fix the gene that causes Rett syndrome, which mainly affects girls, with the hope of helping them feel better and improve their thinking and movement skills.

  NIH research Beckman Research Institute/city of Hope · Duarte, United States · NIH-11142256

• Finding brain signals to help develop treatments for Rett syndrome

  This study is looking at Rett syndrome to find easy ways to measure how well new treatments are working, using both people and mice, so that we can improve future therapies for those affected by this condition.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11103166

• Gene editing to treat Rett syndrome

  This study is exploring new gene editing treatments for Rett syndrome, a condition that mainly affects girls and causes challenges with movement and communication, to see if we can fix the genetic changes that lead to these symptoms and improve their quality of life.

  NIH research Jackson Laboratory · Bar Harbor, United States · NIH-11089360

• Understanding how heat shock responses affect Rett syndrome

  This study is looking at how heat shock signals in the brain might be linked to Rett syndrome, a condition caused by changes in the MeCP2 gene, by comparing brain samples from people with and without the syndrome to find important genes that could help improve diagnosis and treatment for those affected.

  NIH research Loyola University Chicago · Maywood, United States · NIH-11078181

• Developing a treatment for Rett Syndrome using gene therapy techniques

  This study is looking for a new treatment for Rett Syndrome, a condition that mainly affects girls, by using a special approach to help turn on a gene that is important for brain development, with hopes of improving symptoms based on earlier promising results in animals.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11060058

• Understanding early Rett Syndrome development

  This research explores how Rett Syndrome begins in very early development to find new ways to help girls affected by this condition.

  NIH research Massachusetts Institute of Technology · Cambridge, United States · NIH-11009544

• Using a new method to edit genes to treat Rett Syndrome

  This study is exploring a new way to use gene editing to help girls with Rett Syndrome by trying to turn back on a gene that isn’t working properly, and they’ll be testing this method on brain cells grown from Rett patients to see if it’s safe and effective.

  NIH research Epitor Therapeutics, INC. · New York, United States · NIH-11008487

• Improving gene editing in neurons to treat Rett syndrome

  This study is exploring a new way to fix the genetic changes that cause Rett syndrome in brain cells, using a special technique that could lead to better treatments for this condition affecting infants.

  NIH research Univ of Massachusetts Med Sch Worcester · Worcester, United States · NIH-11004385

• Improving brain function in Rett syndrome

  This study is looking at ways to help improve brain function in people with Rett syndrome by exploring how certain gene changes affect brain cells, with the hope of finding new treatments that can boost thinking skills.

  NIH research Brigham and Women's Hospital · Boston, United States · NIH-11001230

• Understanding how specific brain channels affect breathing in Rett syndrome

  This study is looking at how certain brain cells called astrocytes help control breathing in people with Rett syndrome, and it hopes to find out how problems with these cells might affect breathing, which could lead to better understanding and help for those with the condition.

  NIH research University of Connecticut Storrs · Storrs-Mansfield, United States · NIH-10987006

• Understanding motor function and dysfunction in Rett syndrome

  This study is looking at how Rett syndrome affects the brain's ability to control movement, using animal models to understand the problem better, and it aims to find new ways to help those with the condition while also involving students in the research process.

  NIH research University of Arkansas at Fayetteville · Fayetteville, United States · NIH-10974881

• How astrocytes affect Rett syndrome

  This study is looking at how certain brain cells called astrocytes might affect Rett syndrome, and it hopes to find ways to fix protein changes in these cells that could help improve symptoms for people with the condition.

  NIH research Salk Institute for Biological Studies · La Jolla, UNITED STATES · NIH-10953453

• Investigating the role of immune cells in Rett syndrome.

  This study is looking at how certain immune cells from the skull might impact the symptoms of Rett syndrome, a condition that affects brain development, and aims to find new ways to help improve movement and thinking skills for those living with it.

  NIH research Washington University · Saint Louis, United States · NIH-10938021

• Reactivate a gene to treat Rett syndrome

  This study is exploring a new way to help girls with Rett syndrome by trying to turn on a gene that’s important for brain function, using a special method to deliver it to the brain cells that need it, with hopes of improving their symptoms.

  NIH research Columbia University Health Sciences · New York, United States · NIH-10930116

• Investigating ways to reactivate a gene to treat Rett Syndrome

  This study is looking at new ways to help people with Rett Syndrome by using special tools to turn on a gene that isn’t working properly, with the hope of easing their symptoms and improving their lives.

  NIH research Columbia University Health Sciences · New York, United States · NIH-10902891

• Investigating how deep brain stimulation affects memory and motor function in Rett syndrome mice

  This study is looking at how deep brain stimulation might help mice with Rett syndrome by encouraging new brain cell growth and improving their memory and movement skills, which could lead to new treatments for this condition and others like it.

  NIH research Baylor College of Medicine · Houston, United States · NIH-10900594

• Exploring new treatment options for Rett Syndrome

  This study is looking at how Rett Syndrome affects the brain to find new ways to help improve symptoms, especially by focusing on a specific type of brain receptor that could lead to better treatments for girls and women with this condition.

  NIH research Loyola University Chicago · Maywood, United States · NIH-10834181

• Investigating the role of brain cells in Rett syndrome

  This study is looking at how special brain cells, called Bergmann glia, might play a role in the challenges faced by girls with Rett syndrome, and it aims to learn more about how these cells work in mice with a similar genetic change.

  NIH research University of Alabama at Birmingham · Birmingham, United States · NIH-10824337

Related topics

Quick answers

How many Rett Syndrome clinical trials are recruiting right now?

11, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 16 countries. The most sites are in United States (7 trials), Italy (2 trials), Belgium (1 trial), France (1 trial), Georgia (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 2, Phase 1: 1, Phase 2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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