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Showing 1–24 of 57 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Rett Syndrome Clear all
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 Jun 7, 2026, 9:24 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 Jun 7, 2026, 9:24 AM 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 15, 2017 · Synced Jun 7, 2026, 9:24 AM EDT
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 Mar 12, 2026 · Synced Jun 7, 2026, 9:24 AM 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 4, 2021 · Synced Jun 7, 2026, 9:24 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 Jun 7, 2026, 9:24 AM 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 14, 2019 · Synced Jun 7, 2026, 9:24 AM 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 30, 2025 · Synced Jun 7, 2026, 9:24 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 Jun 7, 2026, 9:24 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 Jun 7, 2026, 9:24 AM 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 26, 2025 · Synced Jun 7, 2026, 9:24 AM 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 7, 2022 · Synced Jun 7, 2026, 9:24 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 Jun 7, 2026, 9:24 AM EDT
Completed No phase listed Observational

The Role of Family Functioning in Adaptation to Being a Caregiver of an Individual With Rett Syndrome

NCT00891956
View directory record Official record
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
426 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
3
States / cities
Birmingham, Alabama • Baltimore, Maryland • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Dec 8, 2019 · Synced Jun 7, 2026, 9:24 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
18
States / cities
Birmingham, Alabama • Los Angeles, California • Oakland, California + 15 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2023 · Synced Jun 7, 2026, 9:24 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 Jun 7, 2026, 9:24 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 Jun 7, 2026, 9:24 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 Jun 7, 2026, 9:24 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 Jun 7, 2026, 9:24 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 Jun 7, 2026, 9:24 AM 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 26, 2024 · Synced Jun 7, 2026, 9:24 AM 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 21, 2017 · Synced Jun 7, 2026, 9:24 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 Jun 7, 2026, 9:24 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 Jun 7, 2026, 9:24 AM EDT