Table 1 Shows the summary of the included studies
Study ID | NCT Number | Study Design | Setting | Participants (Inclusion Criteria) | Intervention | Comparison | Dose of Intervention | Conclusion | Follow-up |
|---|---|---|---|---|---|---|---|---|---|
Glaze 2017 | Multicenter randomized controlled trial | USA | Adolescent and adult females with Rett syndrome | Trofinetide | Placebo orally twice a day for 28 days | 35 mg/kg orally twice a day for 28 days or 70 mg/kg orally twice a day for 28 days | Trofinetide at a high dose (70 mg/kg) was more effective than placebo in Rett syndrome | 40 weeks | |
Glaze 2019 | NCT02715115 | Multicenter double-blind placebo-controlled trial | USA | Adolescent or children females with Rett syndrome | Trofinetide | Placebo orally or via a gastrostomy tube twice daily for 42 days | 50 mg/kg, 100 mg/kg, or 200 mg/kg orally or via gastrostomy tube twice daily for 42 days | Trofinetide at a dose of 200 mg/kg bid was tolerable and effective in children with Rett syndrome | 66 days |
Neul 2023 | NCT04181723 | A randomized, parallel-group, placebo-controlled study | USA | Girls and women 5–20 years of age with Rett syndrome | Trofinetide | Placebo orally twice daily for 12 weeks | 200 mg orally twice daily for 12 weeks | Trofinetide was superior to placebo in improving caregiver (RSBQ) and clinician (CGI-I) outcomes | 30 days |