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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

By state

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 NCT07503444

Rett Syndrome Registry

RecruitingObservational study
Ages 0 to 99 ยท All sexes ยท Updated Jun 30, 2026 ยท NCT05432349

Cognitive Function in Rett Syndrome During Trofinetide Treatment

RecruitingObservational study
Ages 18 to 99 ยท All sexes ยท Updated Jun 18, 2026 ยท NCT06705816

Auditory 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 NCT07569445

To 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 NCT06840496

The Rett Syndrome Global Registry

RecruitingObservational study
All ages ยท All sexes ยท Updated Feb 17, 2026 ยท NCT04900493

Neurophysiologic Biomarkers in Rett Syndrome

RecruitingObservational studyHealthy volunteers welcome
Ages 1 to 18 ยท Women ยท Updated Oct 20, 2025 ยท NCT05932589

Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome

Recruiting
Ages 4 to 60 ยท All sexes ยท Updated Oct 3, 2024 ยท NCT05012475

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.