Safety and tolerability of trofinetide was very good at all 3 dose levels. No deaths occurred in the study. Only one participant (200 mg/kg bid group) was withdrawn from the study at the request of her parents because of increased mild gastroesophageal reflux, moderate diarrhea, and mild vomiting, which resolved uneventfully after discontinuation. Four SAEs occurred in 3 participants: 1 participant receiving placebo, 1 participant receiving 100 mg/kg bid, and 1 participant receiving 200 mg/kg bid. All the SAEs were deemed not related to study medication and resolved by the end of the study.
A summary of AEs during the double-blind treatment period occurring in at least 2 participants is shown in table 2. The most common AEs reported during the double-blind period across all treatment groups were diarrhea (27%), vomiting (15%), upper respiratory tract infection (12%), and pyrexia (10%). Diarrhea was reported in 27% in the 50 mg/kg bid group, 13% for the 100 mg/kg bid group, and 56% in the 200 mg/kg bid group. Most AEs were mild or moderate in intensity and most events were considered not related to study drug.
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