Rett Syndrome — Trials, FDA Actions & Pivotal Research

16 active Rett Syndrome trials currently recruiting in the US, sourced from ClinicalTrials.gov.

Synced daily from openFDA and ClinicalTrials.gov. Last sync: September 24, 2026.

Recruiting trials by city

Cities where Rett Syndrome trials in our archive had recruiting sites. Each link lists every currently recruiting paid clinical trial at that location.

Birmingham, AL Fort Collins, CO Little Rock, AR Los Angeles, CA

Active Rett Syndrome trials

Currently recruiting US trials matching Rett Syndrome, sourced live from ClinicalTrials.gov. We’ll add per-event articles here as those trials post results or hit primary completion.

Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome

for RETT Syndrome With Proven MECP2 Mutation

Sponsor: Fenix Innovation Group

Sex: Female·Age: 4 - 25

Code: NCT07257978·Phase 2, Phase 3·Not Yet Recruiting·Listed Dec 2025

To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome

for Rett Syndrome

14 locations incl. Phoenix

Sponsor: Biomed Industries, Inc.

Sex: Female·Age: 5 - 20

Code: NCT06840496·Phase 3·Recruiting·Listed Feb 2025

Apply to this trial

A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome

for Rett Syndrome

Sponsor: Holland Bloorview Kids Rehabilitation Hospital

Sex: Female·Age: 2 - 21

Code: NCT04041713·Phase 2·Recruiting·Listed Aug 2019

Apply to this trial

Adapting a Psychosocial Support Intervention for the Rett Syndrome Community

for Rett Syndrome

1 location in Fort Collins

Sponsor: Colorado State University

Sex: All·Age: 18+

Code: NCT07730112·Not Yet Recruiting·Listed Jul 2026

A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome

for Rett Syndrome

6 locations incl. Little Rock

Sponsor: UCB BIOSCIENCES, Inc.

Sex: All·Age: 5 - 35

Code: NCT07503444·Phase 3·Recruiting·Listed Mar 2026

Apply to this trial

Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome

for Rett Syndrome

Sponsor: Unravel Biosciences, Inc.

Sex: Female·Age: 6 - 21

Code: NCT07150013·Phase 1·Recruiting·Listed Sep 2025

Apply to this trial

Lamivudine as a Novel Clinical Effort for Rett Syndrome

for Rett Syndrome

Sponsor: Maria Denise Fernandes Carvalho de Andrade

Sex: Female·Age: 2+

Code: NCT07640503·Phase 2·Enrolling By Invitation·Listed Jun 2026

Auditory EEG and Behavioral Assessments in Individuals With Rett Syndrome

for Rett Syndrome

1 location in Rochester

Sponsor: University of Rochester

Sex: All·Age: 2+·Accepts healthy volunteers

Code: NCT07569445·Recruiting·Listed May 2026

Apply to this trial

Cognitive Function in Rett Syndrome During Trofinetide Treatment

for Rett Syndrome

1 location in Minneapolis

Sponsor: University of Minnesota

Sex: All·Age: 18 - 99

Code: NCT06705816·Recruiting·Listed Nov 2024

Apply to this trial

Neurophysiologic Biomarkers in Rett Syndrome

for Rett Syndrome

6 locations incl. Los Angeles

Sponsor: Children's Hospital of Philadelphia

Sex: Female·Age: 1 - 18·Accepts healthy volunteers

Code: NCT05932589·Recruiting·Listed Jul 2023

Apply to this trial

Rett Syndrome Registry

for Rett Syndrome

19 locations incl. Birmingham

Sponsor: International Rett Syndrome Foundation

Sex: All·Age: 0 - 99

Code: NCT05432349·Recruiting·Listed Jun 2022

Apply to this trial

The Rett Syndrome Global Registry

for Rett Syndrome

1 location in Trumbull

Sponsor: Rett Syndrome Research Trust

Sex: All·Age: All ages

Code: NCT04900493·Recruiting·Listed May 2021

Apply to this trial

Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome

for Rett Syndrome

1 location in Washington D.C.

Sponsor: Georgetown University

Sex: All·Age: 4 - 60

Code: NCT05012475·Recruiting·Listed Aug 2021

Apply to this trial

Gene Editing as a Therapeutic Approach for Rett Syndrome

for Rett Syndrome

Sponsor: University of Siena

Sex: Female·Age: 6 Months+

Code: NCT05740761·Recruiting·Listed Feb 2023

Apply to this trial

Parent and Infant Inter(X)Action Intervention (PIXI)

for Fragile X Syndrome

1 location in Research Triangle Park

Sponsor: RTI International

Sex: All·Age: Up to 99·Accepts healthy volunteers

Code: NCT03836300·Enrolling By Invitation·Listed Feb 2019

GROWing Up With Rare GENEtic Syndromes

for Prader-Willi Syndrome

Sponsor: dr. Laura C. G. de Graaff-Herder

Sex: All·Age: 18+

Code: NCT04463316·Recruiting·Listed Jul 2020

Apply to this trial