| Recruiting | To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Synd NCT06840496 | Biomed Industries, Inc. | Phase 3 |
| Not Yet Recruiting | Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome NCT07257978 | Fenix Innovation Group | Phase 2 / Phase 3 |
| Not Yet Recruiting | A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome NCT07503444 | UCB BIOSCIENCES, Inc. | Phase 3 |
| Not Yet Recruiting | Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Sy NCT07480564 | Taysha Gene Therapies, Inc. | Phase 3 |
| Not Yet Recruiting | Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome NCT07150013 | Unravel Biosciences, Inc. | Phase 1 |
| Not Yet Recruiting | A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Sy NCT04041713 | Holland Bloorview Kids Rehabilitation Hospital | Phase 2 |
| Active Not Recruiting | Obi Medical Robot: Evaluating Effectiveness Related to Usability NCT07151950 | Desin LLC | — |
| Recruiting | Validating Innovative Biosensors for Rett Autonomic Symptom Tracking NCT06338267 | Rett Syndrome Research Trust | — |
| Recruiting | Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome NCT06856759 | Guangzhou Women and Children's Medical Center | EARLY_Phase 1 |
| Recruiting | Cognitive Function in Rett Syndrome During Trofinetide Treatment NCT06705816 | University of Minnesota | — |
| Active Not Recruiting | Technology-supported Motor Rehabilitation for People With Rett Syndrome NCT07418905 | Georgetown University | N/A |
| Completed | To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome NCT06849973 | Biomed Industries, Inc. | Phase 2 / Phase 3 |
| Active Not Recruiting | Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) NCT06152237 | Taysha Gene Therapies, Inc. | Phase 1 / Phase 2 |
| Active Not Recruiting | Esketamine for the Treatment of Rett Syndrome NCT06199700 | Children's Hospital of Fudan University | EARLY_Phase 1 |
| Recruiting | Neurophysiologic Biomarkers in Rett Syndrome NCT05932589 | Children's Hospital of Philadelphia | — |
| Active Not Recruiting | Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the NCT06621043 | Fenix Innovation Group | Phase 1 / Phase 2 |
| Recruiting | Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) NCT06139172 | Rush University Medical Center | N/A |
| Recruiting | A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome NCT05898620 | Neurogene Inc. | Phase 3 |
| Unknown | The Diagnostic Experience of Male Rett Syndrome NCT06346106 | Children's Hospital Colorado | — |
| Recruiting | A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study) NCT05606614 | Taysha Gene Therapies, Inc. | Phase 3 |
| Unknown | Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Patients NCT05625568 | Vyant Bio | Phase 2 |
| Completed | Osteopathic Manipulative Treatment for Constipation in People With Rett Syndrome NCT05687214 | Ariel University | N/A |
| Recruiting | Rett Syndrome Registry NCT05432349 | International Rett Syndrome Foundation | — |
| Completed | Protective Role of Pre-/ Post-biotics on Gut Inflammation, Dysbiosis, and Life Quality in Rett Syndrome (Bioti NCT05420805 | Azienda Ospedaliera Universitaria Senese | N/A |
| Recruiting | The Rett Syndrome Global Registry NCT04900493 | Rett Syndrome Research Trust | — |
| Terminated | An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome NCT04988867 | ACADIA Pharmaceuticals Inc. | Phase 2 / Phase 3 |
| Completed | Development of the ORCA Communication Measure for Rett Syndrome NCT04920110 | Rett Syndrome Research Trust | — |
| Completed | Tolerability of the Immersive Virtual Reality System Grail in Subjects Affected by Rett Syndrome NCT05691582 | IRCCS Eugenio Medea | N/A |
| Recruiting | Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome NCT05012475 | Georgetown University | N/A |
| Completed | Intensive Anti-Scoliosis Postural Intervention for Individuals With Rett Syndrome Supported by a Smartphone Ap NCT05488938 | Ariel University | N/A |
| Recruiting | Gene Editing as a Therapeutic Approach for Rett Syndrome NCT05740761 | University of Siena | — |
| Completed | ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME NCT04514549 | Rett Syndrome Research Trust | — |
| Terminated | Open-Label Extension Study of Trofinetide for Rett Syndrome NCT04776746 | ACADIA Pharmaceuticals Inc. | Phase 3 |
| Unknown | Biomarkers in Rett Syndrome NCT06346444 | IRCCS Fondazione Stella Maris | — |
| Completed | ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome NCT04304482 | Anavex Life Sciences Corp. | Phase 2 / Phase 3 |
| Terminated | A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome NCT04252586 | Jazz Pharmaceuticals | Phase 3 |
| Completed | Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome NCT04279314 | ACADIA Pharmaceuticals Inc. | Phase 3 |
| Unknown | Telehealth Support to Increase Physical Activity in Rett Syndrome NCT04167059 | Telethon Kids Institute | N/A |
| Completed | Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™) NCT04181723 | ACADIA Pharmaceuticals Inc. | Phase 3 |
| Unknown | Patients With RETT Syndrome NCT04014985 | Assistance Publique Hopitaux De Marseille | N/A |
| Terminated | Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome NCT03848832 | Jazz Pharmaceuticals | Phase 3 |
| Completed | ANAVEX2-73 Study in Patients With Rett Syndrome NCT03941444 | Anavex Life Sciences Corp. | Phase 3 |
| Completed | A Study to Evaluate Ketamine for the Treatment of Rett Syndrome NCT03633058 | Rett Syndrome Research Trust | Phase 2 |
| Completed | Study of ANAVEX2-73 in Patients With Rett Syndrome NCT03758924 | Anavex Life Sciences Corp. | Phase 2 |
| Enrolling By Invitation | Parent and Infant Inter(X)Action Intervention (PIXI) NCT03836300 | RTI International | N/A |
| Enrolling By Invitation | Early Check: Expanded Screening in Newborns NCT03655223 | RTI International | — |
| Recruiting | GROWing Up With Rare GENEtic Syndromes NCT04463316 | dr. Laura C. G. de Graaff-Herder | — |
| Completed | Biobanking of Rett Syndrome and Related Disorders NCT02705677 | University of Alabama at Birmingham | — |
| Unknown | The Role of Probiotics PS128 in Movement Disorders NCT03259971 | National Taiwan University Hospital | N/A |
| Completed | Development of a Behavioral Outcome Measure for Rett Syndrome (RettBe) NCT03196323 | University of South Florida | — |
| Unknown | Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. NCT03059160 | Sheba Medical Center | Phase 2 |
| Completed | 'Uptime' Participation Intervention in Girls and Women With Rett Syndrome NCT03848442 | Rigshospitalet, Denmark | N/A |
| Terminated | Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms NCT02790034 | Newron Pharmaceuticals SPA | Phase 2 / Phase 3 |
| Unknown | Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin NCT02696044 | Center for Rare Neurological Diseases, Norcross, GA | Phase 2 |
| Completed | Assessment of Nutritional and Growth Status in Rett Syndrome Patients NCT06335368 | National Taiwan University Hospital | — |
| Completed | A Safety Study of NNZ-2566 in Pediatric Rett Syndrome NCT02715115 | Neuren Pharmaceuticals Limited | Phase 2 |
| Unknown | The Findings of MR Imaging in Rett Syndrome NCT02903056 | National Taiwan University Hospital | — |
| Completed | Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions NCT02428673 | Gillette Children's Specialty Healthcare | N/A |
| Completed | Natural History of Rett Syndrome & Related Disorders NCT02738281 | University of Alabama at Birmingham | — |
| Terminated | An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome NCT02562820 | The Cleveland Clinic | Phase 1 |
| Completed | Pharmacological Treatment of Rett Syndrome With Statins NCT02563860 | Montefiore Medical Center | Phase 2 |
| Withdrawn | Analysis of the Glutathione Cycle in Children With Rett Syndrome NCT02360436 | Edison Pharmaceuticals Inc | — |
| Unknown | An Open Label, Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls Woth Rett NCT02023424 | Sheba Medical Center | Phase 1 |
| Completed | Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone) NCT02153723 | Montefiore Medical Center | Phase 2 |
| Completed | A Safety Study of NNZ-2566 in Patients With Rett Syndrome NCT01703533 | Neuren Pharmaceuticals Limited | Phase 2 |
| Completed | Phase 2 Study of EPI-743 for Treatment of Rett Syndrome NCT01822249 | Edison Pharmaceuticals Inc | Phase 2 |
| Completed | Treatment of Rett Syndrome With Recombinant Human IGF-1 NCT01777542 | Boston Children's Hospital | Phase 2 |
| Completed | Placebo Controlled Trial of Dextromethorphan in Rett Syndrome NCT01520363 | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Phase 2 |
| Completed | Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW NCT02670694 | Baylor College of Medicine | — |
| Completed | Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection) NCT01253317 | Boston Children's Hospital | Phase 1 |
| Completed | Creatine Metabolism in Rett Syndrome NCT01198015 | Maastricht University Medical Center | — |
| Completed | New Genes Involved in Molecular Etiology of Rett Syndrome Through DNA Microarray Comparative Genomic Hybridiza NCT02885090 | Central Hospital, Nancy, France | N/A |
| Completed | Metabolic Evaluation of Nutrition in Rett Syndrome NCT00786071 | Maastricht University Medical Center | — |
| Completed | The Role of Family Functioning in Adaptation to Being a Caregiver of an Individual With Rett Syndrome NCT00891956 | National Human Genome Research Institute (NHGRI) | — |
| Completed | Pilot Study of the Effects of the Desipramine on the Neurovegetative Parameters of the Child With Rett Syndrom NCT00990691 | Assistance Publique Hopitaux De Marseille | Phase 2 |
| Completed | The Role of Dietary Calcium for the Treatment of Osteopenia in Girls With Rett Syndrome NCT05352373 | Baylor College of Medicine | N/A |
| Completed | Advanced Grandparental Age as a Risk Factor for Autism NCT00464477 | University of Mississippi Medical Center | — |
| Completed | Genetic and Physical Characteristics of Rett Syndrome NCT00299312 | University of Alabama at Birmingham | — |
| Completed | Functional Abilities in Rett Syndrome NCT00630422 | Faculdades Metropolitanas Unidas | — |
| Completed | Effects of Creatine Supplementation in Rett Syndrome NCT01147575 | Medical University of Vienna | N/A |
| Unknown | Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome NCT00069550 | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Phase 3 |
| Terminated | Trial of Dextromethorphan in Rett Syndrome NCT00593957 | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Phase 2 |
| Completed | A Study of the Effectiveness and Safety of Risperidone Versus Placebo in the Treatment of Children With Autist NCT00261508 | Janssen-Ortho Inc., Canada | Phase 3 |
| Completed | Study of Cardiac and Paroxysmal Abnormalities in Rett Syndrome NCT00004773 | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | — |
| Completed | Study of the Pathogenesis of Rett Syndrome NCT00004807 | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | N/A |
| Completed | Nutritional Aspects of Rett Syndrome NCT00004656 | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | N/A |