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ClinicalTrials.gov public records Last synced Oct 26, 2026, 9:14 PM EDT

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Showing 1–24 of 58 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Rett Syndrome Clear all
Recruiting No phase listed Observational Accepts healthy volunteers

Auditory EEG and Behavioral Assessments in Individuals With Rett Syndrome

NCT07569445
View directory record Official record
Conditions
Rett Syndrome
Interventions
Electroencephalogram (EEG) Recording
Procedure
Lead sponsor
University of Rochester
Other
Eligibility
2 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2027
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Oct 26, 2026, 9:14 PM EDT
Completed No phase listed Observational

Natural History of Rett Syndrome & Related Disorders

NCT02738281
View directory record Official record
Conditions
Rett Syndrome, MECP2 Duplication dIsorder, CDKL5 Disorder, FOXG1 Syndrome
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
1,044 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
14
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2021 · Synced Oct 26, 2026, 9:14 PM 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 24, 2005 · Synced Oct 26, 2026, 9:14 PM 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 28, 2024 · Synced Oct 26, 2026, 9:14 PM 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 29, 2023 · Synced Oct 26, 2026, 9:14 PM 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 5, 2021 · Synced Oct 26, 2026, 9:14 PM EDT
Terminated Phase 2 Interventional Results available

Trial of Dextromethorphan in Rett Syndrome

NCT00593957
View directory record Official record
Conditions
Rett Syndrome
Interventions
Dextromethorphan
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
2 Years to 15 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 23, 2014 · Synced Oct 26, 2026, 9:14 PM 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 18, 2026 · Synced Oct 26, 2026, 9:14 PM EDT
Completed No phase listed Observational

Genetic and Physical Characteristics of Rett Syndrome

NCT00299312
View directory record Official record
Conditions
Rett Syndrome, MECP2 Duplication Disorder, Rett-related Disorder
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
11
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2017 · Synced Oct 26, 2026, 9:14 PM 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 17, 2026 · Synced Oct 26, 2026, 9:14 PM EDT
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 27, 2025 · Synced Oct 26, 2026, 9:14 PM EDT
Terminated Phase 3 Interventional Results available

Open-Label Extension Study of Trofinetide for Rett Syndrome

NCT04776746
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
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
21
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 18 more
Source: ClinicalTrials.gov public record
Updated Sep 27, 2024 · Synced Oct 26, 2026, 9:14 PM EDT
Active, not 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
2
States / cities
Chicago, Illinois • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 26, 2026, 9:14 PM EDT
Completed Phase 2 Interventional Results available

Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

NCT01520363
View directory record Official record
Conditions
Rett Syndrome
Interventions
dextromethorphan, placebo
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
1 Year to 10 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 4, 2018 · Synced Oct 26, 2026, 9:14 PM 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 11, 2024 · Synced Oct 26, 2026, 9:14 PM 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 5, 2021 · Synced Oct 26, 2026, 9:14 PM EDT
Completed Phase 1 Interventional Results available

Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)

NCT01253317
View directory record Official record
Conditions
Rett Syndrome
Interventions
rhIGF-1
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
2 Years to 12 Years · Female only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 22, 2017 · Synced Oct 26, 2026, 9:14 PM 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 3, 2022 · Synced Oct 26, 2026, 9:14 PM EDT
Completed No phase listed Observational

Advanced Grandparental Age as a Risk Factor for Autism

NCT00464477
View directory record Official record
Conditions
Autistic Disorder, Pervasive Developmental Disorder, Asperger Syndrome, Childhood Disintegrative Disorder, Rett Syndrome
Interventions
Not listed
Lead sponsor
University of Mississippi Medical Center
Other
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007
U.S. locations
1
States / cities
Jackson, Mississippi
Source: ClinicalTrials.gov public record
Updated Nov 7, 2007 · Synced Oct 26, 2026, 9:14 PM 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 17, 2020 · Synced Oct 26, 2026, 9:14 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

NCT06152237
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
5 Years to 8 Years · Female only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
3
States / cities
Chicago, Illinois • Saint Paul, Minnesota • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 15, 2025 · Synced Oct 26, 2026, 9:14 PM 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 26, 2018 · Synced Oct 26, 2026, 9:14 PM 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 2, 2016 · Synced Oct 26, 2026, 9:14 PM EDT
Completed No phase listed Observational

Predictors of Caregiver Adaptation to Pervasive Developmental Disorders

NCT00496210
View directory record Official record
Conditions
Autism, Asperger's Syndrome, Rett's Disorder
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
324 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 15, 2019 · Synced Oct 26, 2026, 9:14 PM EDT