5 terminated. 5 Phase 3, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 5 | Terminated 5 |
| Phase 2 | 2 | Terminated 2 |
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 |
|---|---|---|---|---|---|---|
| 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 |
| Open-Label Extension Study of Trofinetide for Rett Syndrome trofinetide - Rett Syndrome | Phase 3 | Terminated | ACADIA Pharmaceuticals Inc. | 77 | 2020-11-08 | 2024-09-27 |
| A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome GWP42003-P - Rett Syndrome, RTT | Phase 3 | Terminated | Jazz Pharmaceuticals | 21 | 2020-02-28 | 2022-08-08 |
| Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome GWP42003-P, Placebo - Rett Syndrome, RTT | Phase 3 | Terminated | Jazz Pharmaceuticals | 29 | 2019-07-29 | 2022-09-02 |
| 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 |
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Genetic Diseases, Inborn (5) · Genetic Diseases, X-Linked (5) · Heredodegenerative Disorders, Nervous System (5) · Intellectual Disability (5) · Nervous System Diseases (5) · Neurobehavioral Manifestations (5) · Neurologic Manifestations (5) · X-Linked Intellectual Disability (5)
Cannabidiol (2) · trofinetide (2) · sarizotan (1)
ACADIA Pharmaceuticals Inc. (2) · Jazz Pharmaceuticals (2) · Newron Pharmaceuticals SPA (1)
United States (5) · Italy (3) · United Kingdom (3) · Australia (2) · Spain (2) · Canada (1) · India (1)
Ravi Anand, MD (1)
PMID 37460385, PMID 33862302, PMID 40043705, PMID 42481172
2018 (1), 2019 (1)