Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
Summary
To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome
Timeline
- Start
- 2019-11-06
- Primary completion
- 2021-10-28
- Completion
- 2021-10-28
Outcome
Met primary endpoint
paper LSM Clinical Global Impression-Improvement at week 12 was 3.5 versus 3.8 (P = 0.0030; effect size, 0.47) PMID 37291210 ↗
release “today announced positive top-line results from the pivotal, Phase 3 Lavender™ study evaluating the efficacy and safety of trofinetide” acadia.com ↗
release “including the positive Phase 3 Lavender results” acadia.com ↗
release “The study demonstrated a statistically significant improvement over placebo on the co-primary endpoints” acadia.com ↗
release “at week 12; both were statistically significant” acadia.com ↗
release “While the pivotal LAVENDERTM trial successfully met its co-primary and key secondary endpoints” acadia.com ↗
release “Acadia Pharmaceuticals Announces Positive Top-line Results from the Pivotal Phase 3 Lavender Trial of Trofinetide in Rett Syndrome - Trofinetide met co-primary efficacy endpoints” neurenpharma.com ↗
release “positive top-line results from the pivotal, Phase 3 Lavender™ study evaluating the efficacy and safety of trofinetide” neurenpharma.com ↗
Publications
- Darwish M, Passarell J, Youakim JM, Bradley H, Bishop KM. Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome. Adv Ther. 2024 Apr;41(4):1462-1480. doi: 10.1007/s12325-024-02796-y. Epub 2024 Feb 16.
- Parent H, Ferranti A, Niswender C. Trofinetide: a pioneering treatment for Rett syndrome. Trends Pharmacol Sci. 2023 Oct;44(10):740-741. doi: 10.1016/j.tips.2023.06.008. Epub 2023 Jul 16.
- Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, Lin T, Stankovic S, Bishop KM, Youakim JM. Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study. Nat Med. 2023 Jun;29(6):1468-1475. doi: 10.1038/s41591-023-02398-1. Epub 2023 Jun 8.
- Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Peters SU, Jones NE, Youakim JM. Design and outcome measures of LAVENDER, a phase 3 study of trofinetide for Rett syndrome. Contemp Clin Trials. 2022 Mar;114:106704. doi: 10.1016/j.cct.2022.106704. Epub 2022 Feb 8.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Trofinetide | Peptide | 30 ml | — |
| Subject | Trofinetide | Peptide | 60 ml | — |