Trial reportAdvances in therapy2026
A Phase 1, Randomized, Open-Label Study to Assess the Bioequivalence of Trofinetide as a Ready-to-Use Oral Solution and Constituted Powder for Oral Solution in Healthy Adults.
Trial report in Advances in therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Authors and funding
8 authors.
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Abstract
introductionTrofinetide is an approved treatment in the USA and Canada for Rett syndrome in patients aged ≥ 2 years. Trofinetide is currently supplied as a ready-to-use oral solution (TOS). A new trofinetide powder for oral solution (TPOS) offers potential advantages: smaller configuration of unit-dose packs; excludes preservatives, synthetic red dye, and maltitol; can be dissolved in a range of volumes in cold/room temperature water or water-based beverages; does not require refrigeration. To ensure that the formulation change does not impact trofinetide pharmacokinetics, a bioequivalence study assessed trofinetide pharmacokinetics after single 12,000-mg oral trofinetide doses administered as TOS and TPOS and the impact of a reduced constitution volume on trofinetide bioavailability.
methodsIn this randomized, open-label, three-period, three-treatment design study, 38 healthy adults each received one 12,000-mg trofinetide dose of TOS (60 mL [200 mg/mL]; treatment A), TPOS constituted in 60 mL of water (200 mg/mL; treatment B), and TPOS constituted in 25 mL of water (480 mg/mL; treatment C) in sequence ABC or BAC. Bioequivalence for each treatment comparison was confirmed if the 90% confidence interval (CI) of the geometric mean ratio (GMR) for the maximum observed drug concentration (C
resultsThe criteria for bioequivalence were met for AUC
conclusionThe bioequivalence demonstrated in this study supports the clinical interchangeability of TPOS and TOS and shows that TPOS can be constituted using adjustable volumes of water without affecting bioavailability. There were no unexpected adverse events reported with trofinetide when administered as TPOS and TOS.
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