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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Rett's Disorder Clear all
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 23, 2026, 9:31 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 23, 2026, 9:31 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 23, 2026, 9:31 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

NCT04988867
View directory record Official record
Conditions
Rett Syndrome
Interventions
Trofinetide
Drug
Lead sponsor
ACADIA Pharmaceuticals Inc.
Industry
Eligibility
2 Years to 5 Years · Female only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
7
States / cities
Birmingham, Alabama • Aurora, Colorado • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2024 · Synced Jul 23, 2026, 9:31 AM EDT
Active, not recruiting Not applicable Interventional

Technology-supported Motor Rehabilitation for People With Rett Syndrome

NCT07418905
View directory record Official record
Conditions
Rett Syndrome
Interventions
computer gaming
Behavioral
Lead sponsor
Georgetown University
Other
Eligibility
4 Years to 65 Years · Female only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 19, 2026 · Synced Jul 23, 2026, 9:31 AM EDT
Recruiting Phase 1Phase 2 Interventional

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

NCT06430385
View directory record Official record
Conditions
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Interventions
ION440, Sham procedure
Drug · Procedure
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 65 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
8
States / cities
San Diego, California • Aurora, Colorado • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Jul 23, 2026, 9:31 AM EDT
Completed Phase 2 Interventional Results available

Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)

NCT02153723
View directory record Official record
Conditions
Rett Syndrome
Interventions
Glatiramer Acetate
Drug
Lead sponsor
Montefiore Medical Center
Other
Eligibility
10 Years and older · Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Nov 4, 2018 · Synced Jul 23, 2026, 9:31 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 23, 2026, 9:31 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 23, 2026, 9:31 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 23, 2026, 9:31 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 Jul 23, 2026, 9:31 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 Jul 23, 2026, 9:31 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 23, 2026, 9:31 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 23, 2026, 9:31 AM EDT
Active, not recruiting No phase listed Observational

Obi Medical Robot: Evaluating Effectiveness Related to Usability

NCT07151950
View directory record Official record
Conditions
Upper Extremity Impairments, Cerebral Palsy (CP), ALS - Amyotrophic Lateral Sclerosis, Rett Syndrome
Interventions
One-week trial of Obi Medical device, which provides robotic assistance with feeding to persons with upper extremity impairments who are unable to self-feed in the traditional manner using utensils
Device
Lead sponsor
Desin LLC
Industry
Eligibility
5 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 2, 2025 · Synced Jul 23, 2026, 9:31 AM EDT
Completed Phase 2 Interventional

A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

NCT02715115
View directory record Official record
Conditions
Rett Syndrome
Interventions
NNZ-2566, Placebo
Drug
Lead sponsor
Neuren Pharmaceuticals Limited
Industry
Eligibility
5 Years to 15 Years · Female only
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
12
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2020 · Synced Jul 23, 2026, 9:31 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 Jul 23, 2026, 9:31 AM EDT
Active, not recruiting Phase 3 Interventional

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

NCT05898620
View directory record Official record
Conditions
Rett Syndrome
Interventions
NGN-401
Genetic
Lead sponsor
Neurogene Inc.
Industry
Eligibility
3 Years and older · Female only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
13
States / cities
Birmingham, Alabama • Oakland, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Jul 23, 2026, 9:31 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 23, 2026, 9:31 AM EDT
Completed Not applicable Interventional

Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

NCT02428673
View directory record Official record
Conditions
Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Rett Syndrome
Interventions
Assisted Standing Treatment Program
Other
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
3 Years to 14 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 20, 2021 · Synced Jul 23, 2026, 9:31 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 23, 2026, 9:31 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 23, 2026, 9:31 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 23, 2026, 9:31 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 23, 2026, 9:31 AM EDT