Clinical trials
Rett Syndrome clinical trials
Below are recruiting rett syndrome clinical trials, each written for real people, not researchers. We’re tracking 17 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07503444RecruitingPhase 3
Fenfluramine for Rett syndrome symptoms
This study tests whether fenfluramine, a medication already used for seizures, can help people with Rett syndrome. It aims to see if it improves daily life and reduces symptoms.
Little Rock, ArkansasAges 5–35 - NCT06739434Enrolling by invitation
Gene therapy for Rett syndrome in girls
This trial tests a new gene therapy called GCB-002 for girls with Rett syndrome caused by a MECP2 gene change. It may help improve symptoms by targeting the root genetic cause.
Shanghai, Shanghai MunicipalityAges 2–10 - NCT04900493Recruiting
Rett syndrome global patient and family registry
This registry collects health and family information about people with Rett syndrome (or specific changes in the MECP2 gene). It helps researchers understand the condition better and plan future studies. Participation may include sharing information for living individuals or those who have passed away.
Trumbull, ConnecticutAges Any age - NCT05012475Recruiting
Test whether video games help arm movement in Rett syndrome
This trial looks at whether computer gaming can improve how well you can use your arms to control game actions in Rett syndrome. It checks whether the gaming tasks can be done safely based on your arm movement range.
Washington D.C., District of ColumbiaAges 4–60 - NCT05740761Recruiting
Gene editing for children with Rett syndrome
This trial tests a gene-editing approach for Rett syndrome in children who have a confirmed MECP2 gene mutation. It may help by targeting specific “hotspot” genetic changes that cause the condition.
Siena, SienaAges 6 months+ - NCT05932589Recruiting
Study of brain signals as signs of Rett syndrome
This study measures brain and nerve activity (brain signals) to find patterns that match Rett syndrome. It may help researchers better track Rett changes over time and develop more precise ways to measure response in future treatments.
Los Angeles, CaliforniaAges 1–18 - NCT06338267Recruiting
Testing new sensors for Rett syndrome symptoms at home
This study tests new wearable sensors to track symptoms like heart rate and breathing in people with Rett syndrome. If you join, you'll use the sensors at home to help researchers see how symptoms change day to day.
Chicago, IllinoisAges Any age - NCT06840496RecruitingPhase 3
Study of oral NA-921 vs placebo for girls and women with Rett syndrome
This trial tests an oral medication called NA-921 in females with Rett syndrome to see if it works better than a placebo. It may be an option for those who have a confirmed MECP2 gene mutation and stable seizure patterns.
Phoenix, ArizonaAges 5–20 - NCT07150013RecruitingPhase 1
Vorinostat for Rett syndrome safety study
This trial tests whether vorinostat is safe and helpful for girls and young women with Rett syndrome. Participants must have a confirmed MeCP2 mutation and be in a stable phase of the condition.
MedellínAges 6–21 - NCT05432349Recruiting
Rett syndrome genetic registry for MECP2 changes
This trial is a registry that collects health and genetic information from people with Rett syndrome. It may help researchers better understand how MECP2 loss-of-function changes lead to symptoms and how to support patients.
Birmingham, AlabamaAges birth–99 years - NCT07430046RecruitingPhase 2
Testing mirtazapine for breathing problems in Rett syndrome
This study tests whether the drug mirtazapine can reduce breathing issues in females with Rett syndrome. Participants must have a confirmed MECP2 mutation and specific breathing dysfunction like breath holding or hyperventilation.
GenovaAges 5–40 - NCT07569445Recruiting
Brain activity and hearing tests in Rett syndrome
This study measures brain waves and hearing in people with Rett syndrome to better understand how the condition affects the brain's ability to process sound. The information may help develop better treatments and support.
Rochester, New YorkAges 2 years+ - NCT07640503Enrolling by invitationPhase 2
Lamivudine Treatment Study for Rett Syndrome
This study tests whether lamivudine, a medication originally used for HIV, might help improve symptoms in people with Rett syndrome. Researchers will monitor how safe and effective the treatment is over time.
Fortaleza, CearáAges 2 years+ - NCT04463316Recruiting
Clinic study for people with rare genetic conditions
This study is for people who have a rare syndrome or rare congenital condition and are seen at a rare-disease clinic. It aims to better understand these conditions and how care works in a team setting, which may help guide future treatment decisions.
Rotterdam, South HollandAges 18 years+ - NCT03836300Enrolling by invitation
Parent-infant coaching for families with young diagnoses
This study tests a parent-and-infant coaching program for babies and caregivers. It aims to see whether the program helps after an early diagnosis when English is used at home.
Research Triangle Park, North CarolinaAges Up to 99 years - NCT06705816Recruiting
Cognitive function in Rett syndrome during trofinetide treatment
This study looks at how trofinetide (a medicine for Rett syndrome) affects thinking and memory in people with Rett syndrome. It will follow participants who are starting this treatment through a specific clinic.
Minneapolis, MinnesotaAges 18–99 - NCT07493096Recruiting
Intensive therapy program for children with brain-based conditions
This trial tests an intensive therapy program designed to help children with brain-based conditions like cerebral palsy or autism improve their skills in movement, thinking, and daily activities. The program runs for 2 weeks with daily sessions, and researchers will check for changes in your child's abilities.
The Woodlands, TexasAges 4–12
Hear when a new Rett Syndrome trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.
Rett Syndrome trials by city
Studies with a site in or near these metro areas.
Rett Syndrome trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for rett syndrome?
- Yes. Clin2 currently lists 17 recruiting rett syndrome studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a rett syndrome trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a rett syndrome trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.