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ClinicalTrials.gov public records Last synced Oct 1, 2026, 6:27 PM EDT

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Showing 25–48 of 58 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Rett's Disorder Clear all
Withdrawn No phase listed Observational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 1, 2026, 6:27 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 1, 2026, 6:27 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 1, 2026, 6:27 PM EDT
Terminated Phase 1 Interventional

An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome

NCT02562820
View directory record Official record
Conditions
Rett Syndrome
Interventions
Intravenous infusion
Procedure
Lead sponsor
The Cleveland Clinic
Other
Eligibility
5 Years to 25 Years · Female only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 30, 2017 · Synced Oct 1, 2026, 6:27 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 1, 2026, 6:27 PM 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 14, 2020 · Synced Oct 1, 2026, 6:27 PM 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 21, 2021 · Synced Oct 1, 2026, 6:27 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 1, 2026, 6:27 PM 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 3, 2024 · Synced Oct 1, 2026, 6:27 PM 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
6
States / cities
Little Rock, Arkansas • Miami, Florida • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 1, 2026, 6:27 PM 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 21, 2021 · Synced Oct 1, 2026, 6:27 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 1, 2026, 6:27 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 1, 2026, 6:27 PM 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 24, 2024 · Synced Oct 1, 2026, 6:27 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 1, 2026, 6:27 PM 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 24, 2026 · Synced Oct 1, 2026, 6:27 PM 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 22, 2022 · Synced Oct 1, 2026, 6:27 PM 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 12, 2019 · Synced Oct 1, 2026, 6:27 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 1, 2026, 6:27 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 1, 2026, 6:27 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 1, 2026, 6:27 PM EDT
Terminated Phase 3 Interventional Results available

A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

NCT04252586
View directory record Official record
Conditions
Rett Syndrome, RTT
Interventions
GWP42003-P
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
2 Years to 18 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
14
States / cities
Birmingham, Alabama • La Jolla, California • Aurora, Colorado + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 8, 2022 · Synced Oct 1, 2026, 6:27 PM 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 9, 2026 · Synced Oct 1, 2026, 6:27 PM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Parent and Infant Inter(X)Action Intervention (PIXI)

NCT03836300
View directory record Official record
Conditions
Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
Interventions
Parent-Infant Inter(X)action Intervention (PIXI)
Behavioral
Lead sponsor
RTI International
Other
Eligibility
Up to 99 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2026
U.S. locations
1
States / cities
Research Triangle Park, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 1, 2026, 6:27 PM EDT