Rett Syndrome — Trials, FDA Actions & Pivotal Research

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

Synced daily from openFDA and ClinicalTrials.gov. Last sync: June 12, 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 Chicago, IL Boston, MA La Jolla, CA 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.

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

Apply to this trial

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

A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome

for Rett Syndrome

2 locations incl. Little Rock

Sponsor: UCB BIOSCIENCES, Inc.

Sex: All·Age: 5 - 35

Code: NCT07503444·Phase 3·Not Yet Recruiting

Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome

for Rett Syndrome

1 location in Boston

Sponsor: Taysha Gene Therapies, Inc.

Sex: Female·Age: 2 - 3

Code: NCT07480564·Phase 3·Recruiting

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

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·Not Yet Recruiting

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

Apply to this trial

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

for Rett Syndrome

3 locations incl. Chicago

Sponsor: Rett Syndrome Research Trust

Sex: All·Age: All ages

Code: NCT06338267·Recruiting

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

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

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Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

for Telomeric 22Q13 Monosomy Syndrome

1 location in Chicago

Sponsor: Rush University Medical Center

Sex: All·Age: 2 - 12

Code: NCT06139172·Recruiting

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A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

for Rett Syndrome

13 locations incl. Birmingham

Sponsor: Neurogene Inc.

Sex: Female·Age: 3+

Code: NCT05898620·Phase 3·Recruiting

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A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

for Rett Syndrome

5 locations incl. La Jolla

Sponsor: Taysha Gene Therapies, Inc.

Sex: Female·Age: 6 - 21

Code: NCT05606614·Phase 3·Recruiting

Apply to this trial

Rett Syndrome Registry

for Rett Syndrome

18 locations incl. Birmingham

Sponsor: International Rett Syndrome Foundation

Sex: All·Age: 0 - 99

Code: NCT05432349·Recruiting

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

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

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

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

Early Check: Expanded Screening in Newborns

for Spinal Muscular Atrophy

1 location in Research Triangle Park

Sponsor: RTI International

Sex: All·Age: 1 Day - 31 Days·Accepts healthy volunteers

Code: NCT03655223·Enrolling By Invitation

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

Apply to this trial