Recruiting
Who can join AIPeople with Rett syndrome.
What is being studied AICompares fenfluramine hydrochloride with placebo for effects on symptoms of Rett syndrome.
Study typeTreatment (drug) · Phase 3
CountriesBelgium, France, Germany +6
Rett Syndrome
Browse recruiting and upcoming clinical trials for Rett syndrome.
Patient-friendly, AI-assisted summaries based on official ClinicalTrials.gov ↗ records. Last updated: Oct 10, 2026
About this listHide
ClinicalTrials.gov is the complete and most up-to-date source. Trials may be missing, and trial information may have changed since our last check. Always verify important information in the official registry.
Recruiting
Repurposing Mirtazapine in Rett Syndrome
NCT07430046
Who can join AIGirls and women (female) with Rett syndrome and a confirmed MECP2 mutation, ages 5 to 39, who have frequent daytime breathing problems.
What is being studied AITests mirtazapine oral solution for safety and effects on mood, sleep, motor, and autonomic symptoms.
Study typeTreatment (drug) · Phase 2
CountriesItaly
Recruiting
Who can join AIFemales aged 6–21 years with typical Rett syndrome and a documented disease‑causing MECP2 mutation.
What is being studied AITests safety, tolerability, and transcriptome and clinical changes with oral vorinostat versus placebo.
Study typeTreatment (drug) · Phase 1
CountriesColombia
Recruiting
Who can join AIGirls and women aged 5–20 with classic/typical Rett syndrome and a documented disease-causing MECP2 mutation.
What is being studied AITests oral Bionetide (NA-921) versus placebo in females with Rett syndrome.
Study typeTreatment (drug) · Phase 3
CountriesAustralia, United States
Recruiting
Who can join AIPeople with Rett syndrome ages 4 to 60 who understand cause-and-effect and have functional arm range of motion.
What is being studied AIWhether customized computer games reduce hand stereotypies and increase independent arm/hand use.
Study typeTreatment (other)
CountriesUnited States
Recruiting
Who can join AIFemale children and adolescents (age 2–21) with a diagnosis of Rett syndrome who are at least partially ambulatory.
What is being studied AIWhether Rett‑T (antioxidant cocktail) affects motor function and safety compared with placebo in Rett syndrome.
Study typeTreatment (drug) · Phase 2
CountriesCanada
Not yet recruiting
Who can join AIFemales aged 4–25 years with classical/typical Rett syndrome and a documented pathogenic MECP2 mutation.
What is being studied AICompares NTI164 (low-THC cannabis extract) with placebo for Rett syndrome symptoms.
Study typeTreatment (drug) · Phase 2/3
CountriesAustralia
Enrolling by invitation
Who can join AIFemales aged 2 years or older with a clinical and molecular diagnosis of Rett syndrome who can swallow liquid medication.
What is being studied AITests oral lamivudine for safety and clinical and biological effects in Rett syndrome.
Study typeTreatment (drug) · Phase 2
CountriesBrazil
Enrolling by invitation
Who can join AIPeople with Rett syndrome with a confirmed MECP2 gene mutation.
What is being studied AITests escalating single intrathecal doses of GCB-002 gene therapy.
Study typeTreatment (drug)
CountriesChina
Enrolling by invitation
Who can join AIInfants (15 months or younger) with a confirmed diagnosis of one of several rare neurogenetic conditions and their parent/primary caregiver (English-speaking household).
What is being studied AITests a parent-focused early intervention (PIXI) for infants diagnosed with rare neurogenetic disorders.
Study typeTreatment (other)
CountriesUnited States
Not yet recruiting
Who can join AIAdult family caregivers of individuals with Rett syndrome.
What is being studied AITests an adapted online psychosocial support program for feasibility, acceptability, and preliminary effects.
Study typeOther
CountriesUnited States
Recruiting
Who can join AIPeople with classic Rett syndrome and age-/sex-matched neurologically healthy controls, ages 2 years and older.
What is being studied AICollects EEG and caregiver-reported clinical data to characterize auditory processing in Rett syndrome.
Study typeObservational
CountriesUnited States
Recruiting
Who can join AIChildren aged 4–12 with neurodevelopmental, neurologic, or chromosomal/genetic conditions (for example cerebral palsy, autism, Down syndrome).
What is being studied AICollects real-world before-and-after functional and developmental outcomes from a two-week intensive therapy program.
Study typeObservational
CountriesUnited States
Recruiting
Who can join AIPeople aged 18–99 with Rett syndrome who are starting trofinetide through the Gillette Children's RTT clinic.
What is being studied AIWhether eye-tracking and autonomic measures change during trofinetide treatment.
Study typeObservational
CountriesUnited States
Recruiting
Neurophysiologic Biomarkers in Rett Syndrome
NCT05932589
Who can join AIFemales aged 1–18 years: those with Rett syndrome (including atypical/RTT) and age‑matched typically developing female controls.
What is being studied AICollects EEG and auditory/visual evoked-potential brain measures to identify candidate biomarkers in Rett syndrome.
Study typeObservational
CountriesUnited States
Recruiting
Who can join AIFemale patients with Rett syndrome who have one of four specific MECP2 mutations and are at least 6 months old.
What is being studied AITests CRISPR/Cas9 with AAV delivery to correct common MECP2 mutations.
Study typeObservational
CountriesItaly
Recruiting
GROWing Up With Rare GENEtic Syndromes
NCT04463316
Who can join AIAdults (18+) with complex rare genetic syndromes listed in the study, e.g., Prader‑Willi, Turner, Noonan, Rett and others.
What is being studied AICollects and analyzes adult medical records to describe health problems, labs, symptoms, and medication use.
Study typeObservational
CountriesNetherlands
Recruiting
Rett Syndrome Registry
NCT05432349
Who can join AIPeople of any age with a pathologic loss-of-function alteration in the MECP2 gene, including those with Rett syndrome.
What is being studied AICollects long-term clinical and caregiver-reported data on signs, symptoms, and course of Rett syndrome.
Study typeRegistry
CountriesUnited States
Recruiting
The Rett Syndrome Global Registry
NCT04900493
Who can join AICaregivers of people (any age, living or deceased) with Rett syndrome or an MECP2 mutation.
What is being studied AICollects caregiver-reported health, care, and medical record data about people with Rett syndrome.
Study typeRegistry
CountriesUnited States
Trials that may include Rett syndrome (1)
ClinicalTrials.gov returned these when we searched for this condition, but we could not confirm from the registry records that they are intended for people with it. Check each trial’s eligibility criteria, and ask the study team if you are unsure.
Who can join AIMales aged 2–65 years with genetically confirmed MECP2 Duplication Syndrome.
What is being studied AITests safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal ION440 versus sham.
Study typeTreatment (drug) · Phase 1/2
CountriesAustria, Canada, France +2