48 completed, 12 recruiting, 11 active not recruiting, 10 unknown. 26 Phase 2, 20 Not applicable, 18 Phase 3, 8 Phase 1, 2 Early Phase 1.
Follow Rett Syndrome: new trials by email Literature Review of Rett Syndrome trials Research Report on Rett Syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome NTI164, Placebo - RETT Syndrome With Proven MECP2 Mutation, Rett Syndrome | Phase 2 / Phase 3 | Not yet recruiting | Fenix Innovation Group | 40 | 2027-07-01 | 2026-09-09 |
| To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome Bionetide, Placebo - Rett Syndrome | Phase 3 | Recruiting | Biomed Industries, Inc. | 210 | 2026-09-30 | 2026-03-27 |
| A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome Rett-T, Placebo - Rett Syndrome | Phase 2 | Recruiting | Holland Bloorview Kids Rehabilitation Hospital | 35 | 2026-08-24 | 2026-09-02 |
| Adapting a Psychosocial Support Intervention for the Rett Syndrome Community Those who have patients prescribed Daybue, Caregiver Speaks + Daybue - Rett Syndrome, Family Caregivers | Not applicable | Not yet recruiting | Colorado State University | 60 | 2026-08-01 | 2026-07-28 |
| A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome fenfluramine hydrochloride, Placebo - Rett Syndrome | Phase 3 | Recruiting | UCB BIOSCIENCES, Inc. | 200 | 2026-07-14 | 2026-10-15 |
| Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome TSHA-102 - Rett Syndrome | Phase 3 | Active not recruiting | Taysha Gene Therapies, Inc. | 4 | 2026-05-08 | 2026-08-19 |
| Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome Vorinostat (SAHA), Placebo - Rett Syndrome | Phase 1 | Recruiting | Unravel Biosciences, Inc. | 15 | 2026-03-15 | 2026-09-17 |
| Lamivudine as a Novel Clinical Effort for Rett Syndrome Lamivudine - Rett Syndrome | Phase 2 | Enrolling by invitation | Maria Denise Fernandes Carvalho de Andrade | 10 | 2025-11-05 | 2026-06-10 |
| Auditory EEG and Behavioral Assessments in Individuals With Rett Syndrome Electroencephalogram (EEG) Recording - Rett Syndrome | Recruiting | University of Rochester | 60 | 2025-07-31 | 2026-05-06 | |
| Obi Medical Robot: Evaluating Effectiveness Related to Usability 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 - Upper Extremity Impairments, Cerebral Palsy (CP) | Active not recruiting | Desin LLC | 50 | 2025-05-10 | 2025-09-03 | |
| Validating Innovative Biosensors for Rett Autonomic Symptom Tracking Rett Syndrome | Active not recruiting | Rett Syndrome Research Trust | 30 | 2025-03-03 | 2026-08-31 | |
| Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome Intrathecal injection of AAV-MECP2 for the treatment of Rett syndrome - Rett Syndrome | Early Phase 1 | Active not recruiting | Guangzhou Women and Children's Medical Center | 8 | 2025-01-14 | 2026-05-28 |
| Cognitive Function in Rett Syndrome During Trofinetide Treatment None interventional - Rett Syndrome | Recruiting | University of Minnesota | 20 | 2024-12-01 | 2026-06-18 | |
| 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) ION440, Sham procedure - Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | Phase 1 / Phase 2 | Recruiting | Ionis Pharmaceuticals, Inc. | 48 | 2024-10-21 | 2026-10-08 |
| Technology-supported Motor Rehabilitation for People With Rett Syndrome computer gaming - Rett Syndrome | Not applicable | Active not recruiting | Georgetown University | 14 | 2024-10-01 | 2026-10-28 |
| To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome Drug: Bionetide, Placebo - Rett Syndrome | Phase 2 / Phase 3 | Completed | Biomed Industries, Inc. | 187 | 2024-03-15 | 2025-02-27 |
| Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) TSHA-102 - Rett Syndrome | Phase 1 / Phase 2 | Active not recruiting | Taysha Gene Therapies, Inc. | 6 | 2023-12-12 | 2025-10-15 |
| Esketamine for the Treatment of Rett Syndrome Esketamine hydrochloride - Rett Syndrome | Early Phase 1 | Completed | Children's Hospital of Fudan University | 3 | 2023-11-15 | 2026-05-13 |
| Neurophysiologic Biomarkers in Rett Syndrome EEG and Auditory and Visual Evoked Potentials (AEP and VEP), Clinical assessment - Rett Syndrome, RTT | Recruiting | Children's Hospital of Philadelphia | 222 | 2023-10-11 | 2026-10-26 | |
| Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | Terminated | Ionis Pharmaceuticals, Inc. | 29 | 2023-10-03 | 2026-04-09 |
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Neurologic Manifestations (7) · Autistic Disorder (6) · Genetic Diseases, X-Linked (6) · Angelman Syndrome (5) · Nervous System Diseases (5) · Neurodevelopmental Disorders (5) · Intellectual Disability (4) · Prader-Willi Syndrome (4) · Asperger Syndrome (3) · Autism Spectrum Disorder (3) · Child Development Disorders, Pervasive (3) · Fragile X Syndrome (3) · Mental Disorders (3) · Pathologic Processes (3)
Heredodegenerative Disorders, Nervous System (95) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (93) · Genetic Diseases, Inborn (93) · Neurobehavioral Manifestations (93) · X-Linked Intellectual Disability (92) · Intellectual Disability (91) · Nervous System Diseases (90) · Genetic Diseases, X-Linked (89) · Neurologic Manifestations (88) · Mental Disorders (17) · Pathological Conditions, Signs and Symptoms (13) · Signs and Symptoms (11) · Central Nervous System Diseases (10) · Neurodevelopmental Disorders (9) · Nutritional and Metabolic Diseases (8)
trofinetide (7) · Dextromethorphan (4) · tetrahydro-N, N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride (3) · Cannabidiol (2) · Donepezil (2) · Glatiramer Acetate (2) · Injections (2) · alpha-tocotrienol quinone (2) · mecasermin (2) · triheptanoin (2) · Biomarkers (1) · Blood Specimen Collection (1) · Butyric Acid (1) · Calcium (1) · Creatine (1)
Rett Syndrome Research Trust (5) · ACADIA Pharmaceuticals Inc. (4) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (4) · Anavex Life Sciences Corp. (3) · Edison Pharmaceuticals Inc (3) · National Taiwan University Hospital (3) · Taysha Gene Therapies, Inc. (3) · University of Alabama at Birmingham (3) · Ariel University (2) · Assistance Publique Hopitaux De Marseille (2) · Baylor College of Medicine (2) · Biomed Industries, Inc. (2) · Boston Children's Hospital (2) · Fenix Innovation Group (2) · Georgetown University (2)
United States (55) · Italy (11) · Australia (9) · United Kingdom (7) · Canada (6) · France (6) · Spain (4) · Netherlands (3) · Austria (2) · Brazil (2) · China (2) · Denmark (2) · Israel (2) · Taiwan (2) · Belgium (1)
Alan Percy, MD (4) · Alan K Percy, MD (2) · Barbara B Biesecker (2) · Daniel Glaze, MD (2) · David Nguyen, PhD (2) · Jana von Hehn, PhD (2) · Jeffrey L Neul, MD, PhD (2) · Jeffrey Neul, MD, PhD (2) · Leopold MG Curfs, Professor (2) · Mustafa Sahin, MD, PhD (2) · Timothy Feyma, MD (2) · Aleksandra Djukic, MD PhD (1) · Andreea Nissenkorn, Dr. (1)
PMID 37460385, PMID 38363467, PMID 19772971, PMID 23035069, PMID 24399845, PMID 25071871, PMID 25283752, PMID 25801175, PMID 35568815, PMID 38723617, PMID 15492353, PMID 18337588, PMID 19914908, PMID 20032810, PMID 20231667, PMID 21104896, PMID 21154482, PMID 21637127, PMID 21654506, PMID 22013176, PMID 22174313, PMID 22331013, PMID 22473088, PMID 23696494, PMID 24508304, PMID 26278631, PMID 26995066, PMID 27171548, PMID 27363291, PMID 28132121, PMID 29468173, PMID 31315600, PMID 32165033, PMID 33376177, PMID 33395098, PMID 33407685, PMID 33862302, PMID 35142618, PMID 35149233, PMID 36870948, PMID 37291210, PMID 37833681, PMID 38254921, PMID 38361371, PMID 38917793, PMID 39856538, PMID 40043705, PMID 40173263, PMID 42481172
U54HD061222 (2), 2018 (1), 2019 (1), 2023 (1), 2025 (1), P01HD024448 (1), R33NS130216 (1), R61NS130216 (1)