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Rett Syndrome clinical trials recruiting in the U.S.
9 studies for Rett Syndrome are recruiting participants at sites across the United States.
Rett syndrome is a rare genetic disease. It causes developmental and nervous system problems, mostly in girls. Learn about symptoms and treatments.
Cities with the most studies
- Houston, TX5 studies
- Philadelphia, PA4 studies
- Boston, MA3 studies
- Baltimore, MD3 studies
- Los Angeles, CA3 studies
- Nashville, TN3 studies
- San Diego, CA3 studies
- Aurora, CO3 studies
By state
- California4 studies
- Colorado4 studies
- Florida3 studies
- Maryland3 studies
- Massachusetts4 studies
- Minnesota3 studies
- Pennsylvania4 studies
- Tennessee3 studies
- Texas5 studies
Recently updated studies
A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
RecruitingPhase 3
Ages 5 to 35 ยท All sexes ยท Updated Sep 18, 2026 ยท NCT07503444Rett Syndrome Registry
RecruitingObservational study
Ages 0 to 99 ยท All sexes ยท Updated Jun 30, 2026 ยท NCT05432349Cognitive Function in Rett Syndrome During Trofinetide Treatment
RecruitingObservational study
Ages 18 to 99 ยท All sexes ยท Updated Jun 18, 2026 ยท NCT06705816Auditory EEG and Behavioral Assessments in Individuals With Rett Syndrome
RecruitingObservational studyHealthy volunteers welcome
Ages 2 and older ยท All sexes ยท Updated May 6, 2026 ยท NCT07569445To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
RecruitingPhase 3
Ages 5 to 20 ยท Women ยท Updated Mar 27, 2026 ยท NCT06840496ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
RecruitingPhase 1/2
Ages 2 to 65 ยท Men ยท Updated Mar 24, 2026 ยท NCT06430385The Rett Syndrome Global Registry
RecruitingObservational study
All ages ยท All sexes ยท Updated Feb 17, 2026 ยท NCT04900493Neurophysiologic Biomarkers in Rett Syndrome
RecruitingObservational studyHealthy volunteers welcome
Ages 1 to 18 ยท Women ยท Updated Oct 20, 2025 ยท NCT05932589Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome
Recruiting
Ages 4 to 60 ยท All sexes ยท Updated Oct 3, 2024 ยท NCT05012475From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.