14 completed, 3 terminated, 3 unknown, 2 recruiting. 26 Phase 2, 5 Phase 3, 4 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT) NTI164 - Rett Syndrome | Phase 1 / Phase 2 | Completed | Fenix Innovation Group | 14 | 2023-09-20 | 2026-09-09 |
| Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Patients VYNT-0126, Placebo - Rett Syndrome | Phase 2 | Unknown | Vyant Bio | 48 | 2023-03-01 | 2022-11-23 |
| An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome Trofinetide - Rett Syndrome | Phase 2 / Phase 3 | Terminated | ACADIA Pharmaceuticals Inc. | 15 | 2021-09-22 | 2024-09-24 |
| ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome ANAVEX2-73 oral liquid, Placebo oral liquid - Rett Syndrome | Phase 2 / Phase 3 | Completed | Anavex Life Sciences Corp. | 92 | 2020-07-01 | 2023-08-21 |
| A Study to Evaluate Ketamine for the Treatment of Rett Syndrome Ketamine - Rett Syndrome | Phase 2 | Completed | Rett Syndrome Research Trust | 23 | 2019-03-12 | 2024-05-28 |
| Study of ANAVEX2-73 in Patients With Rett Syndrome ANAVEX2-73, Placebo - Rett Syndrome | Phase 2 | Completed | Anavex Life Sciences Corp. | 31 | 2019-02-28 | 2021-01-12 |
| Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. Tridecanoic Acid - Rett Syndrome | Phase 2 | Unknown | Sheba Medical Center | 10 | 2017-04-01 | 2017-02-23 |
| Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms Sarizotan low dose, Sarizotan high dose, Placebo - Rett Syndrome | Phase 2 / Phase 3 | Terminated | Newron Pharmaceuticals SPA | 129 | 2016-10-26 | 2021-12-21 |
| Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin triheptanoin - Rett Syndrome | Phase 2 | Unknown | Center for Rare Neurological Diseases, Norcross, GA | 12 | 2016-06-01 | 2020-07-17 |
| A Safety Study of NNZ-2566 in Pediatric Rett Syndrome NNZ-2566, Placebo - Rett Syndrome | Phase 2 | Completed | Neuren Pharmaceuticals Limited | 82 | 2016-03-01 | 2020-08-14 |
| Pharmacological Treatment of Rett Syndrome With Statins Lovastatin - Rett Syndrome | Phase 2 | Completed | Montefiore Medical Center | 20 | 2015-07-01 | 2019-07-12 |
| An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder EPI-743 - Autism Spectrum Disorder | Phase 2 | Withdrawn | Edison Pharmaceuticals Inc | 2014-10-31 | 2022-01-21 | |
| Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone) Glatiramer Acetate - Rett Syndrome | Phase 2 | Completed | Montefiore Medical Center | 10 | 2013-08-01 | 2018-11-05 |
| Study to Assess Safety and Efficacy of Fingolimod in Children With Rett Syndrome fingolimod (FTY720) - Rett's Syndrome | Phase 1 / Phase 2 | Completed | University Hospital, Basel, Switzerland | 6 | 2013-08-01 | 2018-06-15 |
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Autistic Disorder (3) · Ataxia (2) · Autism Spectrum Disorder (2) · Angelman Syndrome (1) · Gastrointestinal Diseases (1) · Genetic Diseases, X-Linked (1) · Marijuana Abuse (1) · Mitochondrial Diseases (1) · Neurodevelopmental Disorders (1) · X-Linked Intellectual Disability (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (26) · Genetic Diseases, Inborn (26) · Heredodegenerative Disorders, Nervous System (26) · Intellectual Disability (26) · Nervous System Diseases (26) · Neurobehavioral Manifestations (26) · Neurologic Manifestations (26) · Genetic Diseases, X-Linked (25) · X-Linked Intellectual Disability (25) · Mental Disorders (6) · Child Development Disorders, Pervasive (4) · Neurodevelopmental Disorders (4) · Autism Spectrum Disorder (2) · Dyskinesias (2) · Pathological Conditions, Signs and Symptoms (2)
trofinetide (3) · Dextromethorphan (2) · Injections (2) · alpha-tocotrienol quinone (2) · tetrahydro-N, N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride (2) · triheptanoin (2) · DNA, Ribosomal (1) · Desipramine (1) · Fingolimod Hydrochloride (1) · Glatiramer Acetate (1) · Ketamine (1) · Lamivudine (1) · Lovastatin (1) · Sodium Chloride (1) · Water (1)
Anavex Life Sciences Corp. (2) · Edison Pharmaceuticals Inc (2) · Fenix Innovation Group (2) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (2) · Montefiore Medical Center (2) · Neuren Pharmaceuticals Limited (2) · ACADIA Pharmaceuticals Inc. (1) · Assistance Publique Hopitaux De Marseille (1) · Biomed Industries, Inc. (1) · Boston Children's Hospital (1) · Center for Rare Neurological Diseases, Norcross, GA (1) · Holland Bloorview Kids Rehabilitation Hospital (1) · Ionis Pharmaceuticals, Inc. (1) · Maria Denise Fernandes Carvalho de Andrade (1) · Newron Pharmaceuticals SPA (1)
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Alan Percy, MD (2) · Timothy Feyma, MD (2) · Aleksandra Djukic, MD PhD (1) · Arthur Beisang, MD (1) · Bruria Ben-Zeev, MD (1) · Daniel G Glaze, M.D. (1) · Daniel Glaze, MD (1) · Daniel Tarquinio, DO (1) · David Nguyen, PhD (1) · Eric Marsh, MD (1) · Evdokia Anagnostou, MD (1) · Jana von Hehn, PhD (1) · Jeff Neul, MD (1) · Jeffrey L Neul, M.D., Ph.D. (1) · Jeffrey Neul, MD, PhD (1)
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