Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study
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| Pubblicato in: | Nature Medicine vol. 29, no. 6 (Jun 2023), p. 1468 |
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| Altri autori: | , , , , , , , , |
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Nature Publishing Group
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| Accesso online: | Citation/Abstract Full Text - PDF |
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| 100 | 1 | |a Neul, Jeffrey L. |u Vanderbilt University Medical Center, Vanderbilt Kennedy Center, Nashville, USA (GRID:grid.412807.8) (ISNI:0000 0004 1936 9916) | |
| 245 | 1 | |a Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study | |
| 260 | |b Nature Publishing Group |c Jun 2023 | ||
| 513 | |a Evidence Based Healthcare Journal Article | ||
| 520 | 3 | |a 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 (<ext-link xlink:href="https://clinicaltrials.gov" ext-link-type="url">https://clinicaltrials.gov</ext-link> identifier <ext-link xlink:href="https://clinicaltrials.gov/ct2/show/NCT04181723?term=NCT04181723&draw=2&rank=1" ext-link-type="url">NCT04181723</ext-link>), 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 Impression–Improvement at week 12 was 3.5 versus 3.8 (P = 0.0030; effect size, 0.47). For the key secondary efficacy endpoint, LSM change from baseline to week 12 in the Communication and Symbolic Behavior Scales Developmental Profile Infant–Toddler Checklist Social Composite score was −0.1 versus −1.1 (P = 0.0064; effect size, 0.43). Common treatment-emergent adverse events included diarrhea (80.6% for trofinetide versus 19.1% for placebo), which was mostly mild to moderate in severity. Significant improvement for trofinetide compared with placebo was observed for the coprimary efficacy endpoints, suggesting that trofinetide provides benefit in treating the core symptoms of Rett syndrome.Results from the LAVENDER phase 3 study demonstrate that trofinetide, a synthetic analog of glycine–proline–glutamate, provides significant therapeutic benefits in the core symptoms of Rett syndrome | |
| 653 | |a Neurodevelopmental disorders | ||
| 653 | |a Health services | ||
| 653 | |a Proline | ||
| 653 | |a Glycine | ||
| 653 | |a Growth factors | ||
| 653 | |a Placebos | ||
| 653 | |a Rett syndrome | ||
| 653 | |a Diarrhea | ||
| 653 | |a Effectiveness | ||
| 653 | |a Behavior | ||
| 653 | |a Medicine | ||
| 653 | |a Communication | ||
| 653 | |a Quarantine | ||
| 653 | |a Questionnaires | ||
| 653 | |a Ostomy | ||
| 653 | |a Coronaviruses | ||
| 653 | |a Insulin-like growth factors | ||
| 653 | |a Consent | ||
| 653 | |a Proteins | ||
| 653 | |a Skills | ||
| 653 | |a COVID-19 | ||
| 653 | |a Hospitals | ||
| 653 | |a Social | ||
| 700 | 1 | |a Percy, Alan K. |u University of Alabama at Birmingham, Birmingham, USA (GRID:grid.265892.2) (ISNI:0000000106344187) | |
| 700 | 1 | |a Benke, Timothy A. |u Children’s Hospital of Colorado and University of Colorado School of Medicine, Aurora, USA (GRID:grid.430503.1) (ISNI:0000 0001 0703 675X) | |
| 700 | 1 | |a Berry-Kravis, Elizabeth M. |u Rush University Medical Center, Chicago, USA (GRID:grid.240684.c) (ISNI:0000 0001 0705 3621) | |
| 700 | 1 | |a Glaze, Daniel G. |u Texas Children’s Hospital and Baylor College of Medicine, Houston, USA (GRID:grid.39382.33) (ISNI:0000 0001 2160 926X) | |
| 700 | 1 | |a Marsh, Eric D. |u Children’s Hospital of Philadelphia, Philadelphia, USA (GRID:grid.239552.a) (ISNI:0000 0001 0680 8770) | |
| 700 | 1 | |a Lin, Tim |u Acadia Pharmaceuticals Inc., San Diego, USA (GRID:grid.417646.6) (ISNI:0000 0004 0407 8796) | |
| 700 | 1 | |a Stankovic, Serge |u Acadia Pharmaceuticals Inc., San Diego, USA (GRID:grid.417646.6) (ISNI:0000 0004 0407 8796) | |
| 700 | 1 | |a Bishop, Kathie M. |u Acadia Pharmaceuticals Inc., San Diego, USA (GRID:grid.417646.6) (ISNI:0000 0004 0407 8796) | |
| 700 | 1 | |a Youakim, James M. |u Acadia Pharmaceuticals Inc., San Diego, USA (GRID:grid.417646.6) (ISNI:0000 0004 0407 8796) | |
| 773 | 0 | |t Nature Medicine |g vol. 29, no. 6 (Jun 2023), p. 1468 | |
| 786 | 0 | |d ProQuest |t Science Database | |
| 856 | 4 | 1 | |3 Citation/Abstract |u https://www.proquest.com/docview/2828554641/abstract/embedded/L8HZQI7Z43R0LA5T?source=fedsrch |
| 856 | 4 | 0 | |3 Full Text - PDF |u https://www.proquest.com/docview/2828554641/fulltextPDF/embedded/L8HZQI7Z43R0LA5T?source=fedsrch |