Randomized trial showed trofinetide improved outcomes in girls with RTT, suggesting possible efficacy.
Background/Purpose: Rett syndrome (RTT) is a debilitating genetic neurodevelopmental disorder primarily affecting females. Trofinetide is a synthetic analog of glycine–proline–glutamate, a naturally occurring tripeptide cleaved from insulin-like growth factor 1. Phase 2 studies in RTT demonstrated a clinical benefit over placebo in clinician- and caregiver-assessed efficacy measures. Here, we present the results of LAVENDER, a randomized, placebo-controlled, phase 3 study of trofinetide in girls and young women with RTT.
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Huber et al. (2025) studied this question.
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