At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 187 enrolled
Drug / intervention
Trofinetide +1 moredrug
Likely dose
Trofinetide solution administered based on subject's weight at Baseline, twice dailyAI-extracted
Key inclusion· 7
- ✓Female aged 5 to 20 years at Screening
- ✓Body weight ≥12 kg at Screening
- ✓Can swallow liquid medication or receive via gastrostomy tube
- ✓Classic/typical Rett syndrome diagnosis
Key exclusion· 6
- ✕Insulin treatment within 12 weeks of Baseline
- ✕Clinically significant cardiovascular, endocrine, renal, hepatic, respiratory or gastrointestinal disease
- ✕History of or current cerebrovascular disease or brain trauma
- ✕Significant uncorrected visual or hearing impairment
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
In Brief
A Phase 3 clinical trial evaluating Trofinetide and Placebo for Rett Syndrome. Completed, enrolled 187 participants across 21 sites.
Detailed Summary
To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsRett Syndrome
CountriesUnited States
Collaborators--
Timeline
Phase 3CompletedFinished
2020202120222023202420252026
Enrollment StartNov 2019
First PostedNov 2019
Primary CompletionOct 2021
TodayJul 2026
First PostedNov 29, 2019
Enrollment StartNov 6, 2019
Primary CompletionOct 28, 2021
TodayJul 2, 2026
Enrollment to primary: 2.0 yearsPosted 6.6 years ago
Interventions
Trofinetidedrug
Trofinetide solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Placeboother
Trofinetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks