Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study
Vanderbilt University Medical Center · University of Alabama at Birmingham · +7 more institutions
Abstract
Rett syndrome is a rare, genetic neurodevelopmental disorder. Trofinetide is a synthetic analog of glycine-proline-glutamate, the N-terminal tripeptide of the insulin-like growth factor 1 protein, and has demonstrated clinical benefit in phase 2 studies in Rett syndrome. In this phase 3 study ( https://clinicaltrials.gov identifier NCT04181723 ), females with Rett syndrome received twice-daily oral trofinetide (n = 93) or placebo (n = 94) for 12 weeks. For the coprimary efficacy endpoints, least squares mean (LSM) change from baseline to week 12 in the Rett Syndrome Behaviour Questionnaire for trofinetide versus placebo was -4.9 versus -1.7 (P = 0.0175; Cohen's d effect size, 0.37), and LSM Clinical Global…
Citation impact
- FWCI
- 52.92
- Percentile
- 100%
- References
- 43
Authors
10- JLJeffrey L. NeulCorresponding
Vanderbilt University Medical Center
- AKAlan K. Percy
University of Alabama at Birmingham
- TATim A. Benke
Children's Hospital Colorado, University of Colorado Denver
- EBElizabeth Berry‐Kravis
Rush University Medical Center
- DGDaniel G. Glaze
Baylor College of Medicine, Texas Children's Hospital
Topics & keywords
- Rett syndrome
- Placebo
- Medicine
- Internal medicine
- Clinical endpoint
- Pediatrics
- Clinical trial
- Pathology
Funding
- SFSimons Foundation
- IRInternational Rett Syndrome Foundation
- BBiogen
- CHChildren's Hospital of Philadelphia
- VUVanderbilt University
- UOUniversity of Pennsylvania
- EUEmory University
- TCTexas Children's Hospital
- CCCincinnati Children's Hospital Medical Center
- CHChildren's Hospital Colorado
- CCCleveland Clinic
- CTClinical Trial Center, China Medical University Hospital
- UOUniversity of Cincinnati
- RURush University
- UOUniversity of South Florida
- MIMIND Institute, University of California, Davis
- ZZogenix
- VUVanderbilt University Medical Center
- UPUltragenyx Pharmaceutical
- IPIonis Pharmaceuticals
- GPGW Pharmaceuticals
- APACADIA Pharmaceuticals
- MPMallinckrodt Pharmaceuticals
- ZPZynerba Pharmaceuticals
- AAveXis
- MPMarinus Pharmaceuticals
- LFLouLou Foundation
- IFInternational Foundation for CDKL5 Research
- NINational Institutes of Health
- UOUniversity of California, Davis
- UOUniversity of North Carolina at Chapel Hill
- UOUniversity of California, San Diego
- NINational Institute of Neurological Disorders and Stroke
- NINational Institute of Child Health and Human Development