Trial reportClinical drug investigation2022
A Phase 1, Open-Label Study to Evaluate the Effects of Food and Evening Dosing on the Pharmacokinetics of Oral Trofinetide in Healthy Adult Subjects.
Trial report in Clinical drug investigation, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
11 citing papers in PubMed, 1 synthesis or guideline pooled it, 21 citations in OpenAlex.
- Is trofinetide a future treatment for Rett syndrome? A comprehensive systematic review and meta-analysis of randomized controlled trials.BMC medicine · 2024Pooled it
- 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.Advances in therapy · 2026Trial
- Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome.Advances in therapy · 2024Trial
- Population Pharmacokinetics of Trofinetide in a Pediatric Population Aged 2-4 Years with Rett Syndrome.Advances in therapy · 2025Article
- Population Pharmacokinetic Modeling to Support Trofinetide Dosing for the Treatment of Rett Syndrome.Advances in therapy · 2025Article
- Effect of Hepatic Impairment on Trofinetide Exposures Using an In Silico Physiologically Based Pharmacokinetic Model.Advances in therapy · 2024Article
- Trofinetide receives FDA approval as first drug for Rett syndrome.Annals of medicine and surgery (2012) · 2024Article
- Characterization of the Pharmacokinetics and Mass Balance of a Single Oral Dose of Trofinetide in Healthy Male Subjects.Clinical drug investigation · 2024Article
- Trofinetide for Rett Syndrome: Highlights on the Development and Related Inventions of the First USFDA-Approved Treatment for Rare Pediatric Unmet Medical Need.Journal of clinical medicine · 2023Review
- Trofinetide: First Approval.Drugs · 2023Review
- Cyclic Glycine-Proline (cGP) Normalises Insulin-Like Growth Factor-1 (IGF-1) Function: Clinical Significance in the Ageing Brain and in Age-Related Neurological Conditions.Molecules (Basel, Switzerland) · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
objectiveTrofinetide, a synthetic analog of tripeptide glycine-proline-glutamate, is an investigational agent for the treatment of Rett syndrome, a neurodevelopmental disorder with affected individuals requiring lifelong support. Food can affect the pharmacokinetic profile of a drug, and this phase 1 study assessed the potential effect of food on the pharmacokinetics of trofinetide. The study also evaluated the potential effect of evening dosing on trofinetide bioavailability and characterized the pharmacokinetic profile of trofinetide in urine.
methodsA 60 mL oral solution of trofinetide (12 g) was administered in three dosing periods: morning fasted (A; reference), morning fed (B), and evening fasted (C). Healthy adult subjects (18-45 years) were randomized to sequence ABC (n = 19) or BAC (n = 22). Blood and urine samples were collected at scheduled timepoints for trofinetide pharmacokinetic analysis. Bioequivalence was confirmed if 90% confidence intervals for geometric mean ratio between B/A or C/A fell within 80-125% equivalence limits for area under the concentration-time curve (AUC) and maximum concentration (C
resultsBioequivalence criteria were met for all conditions (i.e., morning fed vs. morning fasted and evening fasted vs. morning fasted) except C
conclusionThis study supports dosing of trofinetide without regard to food.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.