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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 3 Interventional Results available

Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)

NCT04181723
View directory record Official record
Conditions
Rett Syndrome
Interventions
Trofinetide, Placebo
Drug · Other
Lead sponsor
ACADIA Pharmaceuticals Inc.
Industry
Eligibility
5 Years to 20 Years · Female only
Enrollment
187 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
21
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 18 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2024 · Synced Sep 7, 2026, 1:51 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 Sep 7, 2026, 1:51 AM EDT
Completed No phase listed Observational

ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME

NCT04514549
View directory record Official record
Conditions
Rett Syndrome
Interventions
Emerald
Device
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
Female only
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced Sep 7, 2026, 1:51 AM EDT
Terminated No phase listed Observational

Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

NCT06014541
View directory record Official record
Conditions
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
1 Month to 65 Years · Male only
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
5
States / cities
San Diego, California • Saint Paul, Minnesota • Philadelphia, Pennsylvania + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Sep 7, 2026, 1:51 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 Sep 7, 2026, 1:51 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 Sep 7, 2026, 1:51 AM EDT
Not yet recruiting Not applicable Interventional

Adapting a Psychosocial Support Intervention for the Rett Syndrome Community

NCT07730112
View directory record Official record
Conditions
Rett Syndrome, Family Caregivers, Social Support, Psychological Support
Interventions
Those who have patients prescribed Daybue, Caregiver Speaks + Daybue
Other
Lead sponsor
Colorado State University
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Fort Collins, Colorado
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Sep 7, 2026, 1:51 AM EDT
Recruiting No phase listed Observational

Rett Syndrome Registry

NCT05432349
View directory record Official record
Conditions
Rett Syndrome, Rett Syndrome, Atypical, Genetic Disease, Genetic Diseases, X-Linked, Intellectual Disability, Neurobehavioral Manifestations, Neurologic Manifestations, Neurologic Disorder, Neurodevelopmental Disorders, Nervous System Diseases
Interventions
Not listed
Lead sponsor
International Rett Syndrome Foundation
Other
Eligibility
0 Years to 99 Years
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
19
States / cities
Birmingham, Alabama • Los Angeles, California • Oakland, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Sep 7, 2026, 1:51 AM 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 3, 2018 · Synced Sep 7, 2026, 1:51 AM EDT
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 – 2031
U.S. locations
5
States / cities
Little Rock, Arkansas • Orlando, Florida • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 16, 2026 · Synced Sep 7, 2026, 1:51 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 Sep 7, 2026, 1:51 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 Sep 7, 2026, 1:51 AM 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 14, 2025 · Synced Sep 7, 2026, 1:51 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 Sep 7, 2026, 1:51 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 Sep 7, 2026, 1:51 AM EDT
Completed Phase 2 Interventional

Study of ANAVEX2-73 in Patients With Rett Syndrome

NCT03758924
View directory record Official record
Conditions
Rett Syndrome
Interventions
ANAVEX2-73, Placebo
Drug
Lead sponsor
Anavex Life Sciences Corp.
Industry
Eligibility
18 Years to 45 Years · Female only
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
8
States / cities
Birmingham, Alabama • Sacramento, California • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Jan 11, 2021 · Synced Sep 7, 2026, 1:51 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 Sep 7, 2026, 1:51 AM EDT
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 5, 2026 · Synced Sep 7, 2026, 1:51 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms

NCT02790034
View directory record Official record
Conditions
Rett Syndrome
Interventions
Sarizotan low dose, Sarizotan high dose, Placebo
Drug
Lead sponsor
Newron Pharmaceuticals SPA
Industry
Eligibility
4 Years and older
Enrollment
129 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
5
States / cities
Birmingham, Alabama • San Diego, California • Chicago, Illinois + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 20, 2021 · Synced Sep 7, 2026, 1:51 AM 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 30, 2026 · Synced Sep 7, 2026, 1:51 AM 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 6, 2007 · Synced Sep 7, 2026, 1:51 AM EDT
Active, not recruiting Not applicable Interventional

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

NCT06139172
View directory record Official record
Conditions
Telomeric 22Q13 Monosomy Syndrome, Tuberous Sclerosis, Hamartoma Syndrome, Multiple, Fragile X Syndrome, Angelman Syndrome, Rett Syndrome, Chromosome 15Q, Partial Deletion, Creatine Deficiency, X-linked
Interventions
Functional Behavioral Training (FBT), Positive Parenting Strategies-Treatment as Usual
Behavioral · Other
Lead sponsor
Rush University Medical Center
Other
Eligibility
2 Years to 12 Years
Enrollment
92 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 7, 2026, 1:51 AM 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 22, 2014 · Synced Sep 7, 2026, 1:51 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 Sep 7, 2026, 1:51 AM EDT