3 completed, 2 recruiting, 1 active not recruiting, 1 terminated. 8 Phase 1, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 8 | Completed 3, Recruiting 2, Active not recruiting 1, Terminated 1, Unknown 1 |
| Phase 2 | 4 | Completed 2, Active not recruiting 1, Recruiting 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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome Intravenous infusion - Rett Syndrome | Phase 1 | Terminated | The Cleveland Clinic | 4 | 2015-11-01 | 2017-08-30 |
| An Open Label, Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls Woth Rett Syndrome Glatiramer Acetate (Copaxone®) - Rett Syndrome | Phase 1 | Unknown | Sheba Medical Center | 10 | 2014-01-01 | 2014-02-04 |
| 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 |
| Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection) rhIGF-1 - Rett Syndrome | Phase 1 | Completed | Boston Children's Hospital | 12 | 2010-12-01 | 2017-06-22 |
Neurodevelopmental Disorders (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Genetic Diseases, Inborn (8) · Genetic Diseases, X-Linked (8) · Heredodegenerative Disorders, Nervous System (8) · Intellectual Disability (8) · Nervous System Diseases (8) · Neurobehavioral Manifestations (8) · Neurologic Manifestations (8) · X-Linked Intellectual Disability (8) · Mental Disorders (1)
Fingolimod Hydrochloride (1) · Glatiramer Acetate (1) · Infusions, Intravenous (1) · Vorinostat (1) · mecasermin (1)
Boston Children's Hospital (1) · Fenix Innovation Group (1) · Ionis Pharmaceuticals, Inc. (1) · Sheba Medical Center (1) · Taysha Gene Therapies, Inc. (1) · The Cleveland Clinic (1) · University Hospital, Basel, Switzerland (1) · Unravel Biosciences, Inc. (1)
United States (4) · Canada (2) · Australia (1) · Austria (1) · Colombia (1) · France (1) · Israel (1) · Spain (1) · Switzerland (1) · United Kingdom (1)
Andreea Nissenkorn, Dr. (1) · Bruria Ben Zeev, prof. (1) · Daniel Sessler, MD (1) · Irit Avisar, R.N M.A (1) · Laura Pisani, M.D. (1) · Ludwig Kappos, Prof. (1) · Mustafa Sahin, MD, PhD (1) · Neal I Muni, M.D., MSPH (1)
2023 (1)