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Showing 1–24 of 57 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 2Phase 3 Interventional

To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome

NCT06849973
View directory record Official record
Conditions
Rett Syndrome
Interventions
Drug: Bionetide, Placebo
Drug
Lead sponsor
Biomed Industries, Inc.
Industry
Eligibility
5 Years to 20 Years · Female only
Enrollment
187 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
12
States / cities
La Jolla, California • Los Angeles, California • Sacramento, California + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2025 · Synced Jul 21, 2026, 11:18 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Rare Diseases Clinical Research Network: Neurophysiological Correlates

NCT03077308
View directory record Official record
Conditions
Rett Syndrome, Preserved Speech Variant, Mecp2 Duplication Syndrome, Rett-related Disorders
Interventions
Auditory and Visual Event-related Potentials and EEG
Procedure
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
2 Years to 65 Years
Enrollment
185 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2021
U.S. locations
5
States / cities
Denver, Colorado • Boston, Massachusetts • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced Jul 21, 2026, 11:18 AM EDT
Completed Phase 2 Interventional

A Safety Study of NNZ-2566 in Patients With Rett Syndrome

NCT01703533
View directory record Official record
Conditions
Rett Syndrome
Interventions
NNZ-2566, Placebo
Drug
Lead sponsor
Neuren Pharmaceuticals Limited
Industry
Eligibility
16 Years to 45 Years · Female only
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
3
States / cities
Birmingham, Alabama • Saint Paul, Minnesota • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 4, 2018 · Synced Jul 21, 2026, 11:18 AM EDT
Terminated Phase 3 Interventional Results available

Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

NCT03848832
View directory record Official record
Conditions
Rett Syndrome, RTT
Interventions
GWP42003-P, Placebo
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
2 Years to 18 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
14
States / cities
Birmingham, Alabama • San Diego, California • Aurora, Colorado + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2022 · Synced Jul 21, 2026, 11:18 AM EDT
Completed No phase listed Observational

Studies in Patients With Tuberous Sclerosis Complex

NCT03276195
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
Translational Genomics Research Institute
Other
Eligibility
Not listed
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 28, 2023 · Synced Jul 21, 2026, 11:18 AM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

NCT03633058
View directory record Official record
Conditions
Rett Syndrome
Interventions
Ketamine
Drug
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
6 Years to 12 Years · Female only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
7
States / cities
Birmingham, Alabama • Aurora, Colorado • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated May 27, 2024 · Synced Jul 21, 2026, 11:18 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Development of the ORCA Communication Measure for Rett Syndrome

NCT04920110
View directory record Official record
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
18 Years and older
Enrollment
272 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 21, 2022 · Synced Jul 21, 2026, 11:18 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Neurophysiologic Biomarkers in Rett Syndrome

NCT05932589
View directory record Official record
Conditions
Rett Syndrome, RTT, Rett Syndrome, Atypical
Interventions
EEG and Auditory and Visual Evoked Potentials (AEP and VEP), Clinical assessment
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
1 Year to 18 Years · Female only
Enrollment
202 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2029
U.S. locations
6
States / cities
Los Angeles, California • Aurora, Colorado • Brookline, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2025 · Synced Jul 21, 2026, 11:18 AM EDT
Not yet recruiting Phase 3 Interventional

A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome

NCT07503444
View directory record Official record
Conditions
Rett Syndrome
Interventions
fenfluramine hydrochloride, Placebo
Drug · Other
Lead sponsor
UCB BIOSCIENCES, Inc.
Industry
Eligibility
5 Years to 35 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
4
States / cities
Little Rock, Arkansas • Orlando, Florida • Houston, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Jul 21, 2026, 11:18 AM EDT
Recruiting Phase 3 Interventional

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

NCT07480564
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
2 Years to 3 Years · Female only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Jul 21, 2026, 11:18 AM EDT
Not listed Phase 3 Interventional

Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome

NCT00069550
View directory record Official record
Conditions
Rett Syndrome
Interventions
dextromethorphan, donepezil hydrochloride
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Year to 15 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 21, 2026, 11:18 AM EDT
Completed No phase listed Observational

Biobanking of Rett Syndrome and Related Disorders

NCT02705677
View directory record Official record
Conditions
Rett Syndrome, MECP2 Duplication, CDKL5, FOXG1 Disorders
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
752 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
12
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced Jul 21, 2026, 11:18 AM EDT
Completed Phase 2 Interventional Results available

Pharmacological Treatment of Rett Syndrome With Statins

NCT02563860
View directory record Official record
Conditions
Rett Syndrome
Interventions
Lovastatin
Drug
Lead sponsor
Montefiore Medical Center
Other
Eligibility
3 Years and older · Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Jul 11, 2019 · Synced Jul 21, 2026, 11:18 AM EDT
Completed Phase 3 Interventional Results available

Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

NCT04279314
View directory record Official record
Conditions
Rett Syndrome
Interventions
Trofinetide
Drug
Lead sponsor
ACADIA Pharmaceuticals Inc.
Industry
Eligibility
5 Years to 21 Years · Female only
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
21
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 18 more
Source: ClinicalTrials.gov public record
Updated Apr 10, 2024 · Synced Jul 21, 2026, 11:18 AM EDT
Withdrawn No phase listed Observational

Analysis of the Glutathione Cycle in Children With Rett Syndrome

NCT02360436
View directory record Official record
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
Edison Pharmaceuticals Inc
Industry
Eligibility
2 Years to 10 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Feb 2, 2022 · Synced Jul 21, 2026, 11:18 AM EDT
Recruiting No phase listed Observational

The Rett Syndrome Global Registry

NCT04900493
View directory record Official record
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
1
States / cities
Trumbull, Connecticut
Source: ClinicalTrials.gov public record
Updated Feb 16, 2026 · Synced Jul 21, 2026, 11:18 AM EDT
Active, not recruiting Phase 3 Interventional

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

NCT05606614
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
6 Years to 21 Years · Female only
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
8
States / cities
Duarte, California • La Jolla, California • Oakland, California + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 21, 2026, 11:18 AM EDT
Not listed No phase listed Observational

The Diagnostic Experience of Male Rett Syndrome

NCT06346106
View directory record Official record
Conditions
Rett Syndrome, RTT
Interventions
Male Rett
Genetic
Lead sponsor
Children's Hospital Colorado
Other
Eligibility
0 Years to 100 Years · Male only
Enrollment
80 participants
Timeline
2023 – 2024
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Apr 2, 2024 · Synced Jul 21, 2026, 11:18 AM EDT
Recruiting No phase listed Observational

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

NCT06338267
View directory record Official record
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
3
States / cities
Chicago, Illinois • Boston, Massachusetts • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Jul 21, 2026, 11:18 AM EDT
Not listed Phase 2 Interventional

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

NCT02696044
View directory record Official record
Conditions
Rett Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Center for Rare Neurological Diseases, Norcross, GA
Other
Eligibility
2 Years and older · Female only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Norcross, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 16, 2020 · Synced Jul 21, 2026, 11:18 AM EDT
Completed Phase 2 Interventional Results available

Treatment of Rett Syndrome With Recombinant Human IGF-1

NCT01777542
View directory record Official record
Conditions
Rett Syndrome
Interventions
Recombinant Human Insulin Growth Factor 1 (rhIGF-1), Placebo
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
2 Years to 10 Years · Female only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 25, 2018 · Synced Jul 21, 2026, 11:18 AM EDT
Completed No phase listed Observational

Development of a Behavioral Outcome Measure for Rett Syndrome (RettBe)

NCT03196323
View directory record Official record
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
3 Years to 18 Years · Female only
Enrollment
146 participants
Timeline
2017 – 2020
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Dec 8, 2020 · Synced Jul 21, 2026, 11:18 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW

NCT02670694
View directory record Official record
Conditions
Rett Syndrome, Prader-Willi Syndrome, Angelman Syndrome, Sleep Problems
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Year to 18 Years
Enrollment
804 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
9
States / cities
Birmingham, Alabama • Irvine, California • San Diego, California + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2016 · Synced Jul 21, 2026, 11:18 AM EDT
Recruiting No phase listed Observational

Cognitive Function in Rett Syndrome During Trofinetide Treatment

NCT06705816
View directory record Official record
Conditions
Rett Syndrome
Interventions
None interventional
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 99 Years
Enrollment
20 participants
Timeline
2024 – 2027
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Jul 21, 2026, 11:18 AM EDT