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.

Darwish M, Yamamoto A, Adegbenle Y, Tejwani R, Chen Y, Nunez-Disla R, Youakim JM, Pathak S

Advances in therapy12 Feb 2026

PMID41678137PMCIDPMC13065568DOI10.1007/s12325-025-03484-1PepBankPUB-41678137

Read at source PubMedAsk about this study

Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: CC BY-NC. Results still quote short attributed sentences.

Results from this study12

12 results from studies that gave the peptide. 12 results from people.

01HumanRandomized controlled trialPharmacokinetics
In healthy adults, trofinetide powder in 60 mL water was bioequivalent to ready-to-use solution for AUC0-t (GMR 99.62%, CI 96.52-102.83).
In plain wordsIn healthy adults, trofinetide powder in 60 mL water and ready-to-use solution had equivalent AUC0-t.
Peptide
Trofinetide
Outcome
No significant changeAUC0-t bioequivalence: B versus A
Study
n = 38
Comparator
ready-to-use oral solution (A)
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
12,000 mg · oral · single doseAs given in this study (human).
Measurements
AUC0-t GMR
99.62%
B versus A
90% CI
96.52 to 102.83%
B versus A
Source sentences
Summary
The criteria for bioequivalence were met for AUC_0-t (GMR 99.62%; CI 96.52-102.83), AUC_0-∞ (GMR 99.50%; CI 96.44-102.66), and C_max (GMR 99.52%; CI 94.99-104.27) between treatment B and treatment A.
1 context sentence(design, population, supporting numbers)
Summary · context
In 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; treatmen…
Report a problemFND-4167813701
02HumanRandomized controlled trialPharmacokinetics
In healthy adults, trofinetide powder in 60 mL water was bioequivalent to ready-to-use solution for AUC0-∞ (GMR 99.50%, CI 96.44-102.66).
In plain wordsIn healthy adults, trofinetide powder in 60 mL water and ready-to-use solution had equivalent total exposure to infinity.
Peptide
Trofinetide
Outcome
No significant changeAUC0-infinity bioequivalence: B versus A
Study
n = 38
Comparator
ready-to-use oral solution (A)
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
12,000 mg · oral · single doseAs given in this study (human).
Measurements
AUC0-infinity GMR
99.5%
B versus A
90% CI
96.44 to 102.66%
B versus A
Source sentences
Summary
The criteria for bioequivalence were met for AUC_0-t (GMR 99.62%; CI 96.52-102.83), AUC_0-∞ (GMR 99.50%; CI 96.44-102.66), and C_max (GMR 99.52%; CI 94.99-104.27) between treatment B and treatment A.
1 context sentence(design, population, supporting numbers)
Summary · context
In 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; treatmen…
Report a problemFND-4167813702
03HumanRandomized controlled trialPharmacokinetics
In healthy adults, trofinetide powder in 60 mL water was bioequivalent to ready-to-use solution for Cmax (GMR 99.52%, CI 94.99-104.27).
In plain wordsIn healthy adults, trofinetide powder in 60 mL water and ready-to-use solution had equivalent peak concentrations.
Peptide
Trofinetide
Outcome
No significant changeCmax bioequivalence: B versus A
Study
n = 38
Comparator
ready-to-use oral solution (A)
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
12,000 mg · oral · single doseAs given in this study (human).
Measurements
Cmax GMR
99.52%
B versus A
90% CI
94.99 to 104.27%
B versus A
Source sentences
Summary
The criteria for bioequivalence were met for AUC_0-t (GMR 99.62%; CI 96.52-102.83), AUC_0-∞ (GMR 99.50%; CI 96.44-102.66), and C_max (GMR 99.52%; CI 94.99-104.27) between treatment B and treatment A.
1 context sentence(design, population, supporting numbers)
Summary · context
In 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; treatmen…
Report a problemFND-4167813703
04HumanNon-randomized trialPharmacokinetics
In healthy adults, trofinetide powder in 25 mL water met AUC0-t bioequivalence criteria versus ready-to-use solution.
In plain wordsIn healthy adults, trofinetide powder in 25 mL water and ready-to-use solution had equivalent AUC0-t.
Peptide
Trofinetide
Outcome
No significant changeAUC0-t bioequivalence: C versus A
Study
n = 38
Comparator
ready-to-use oral solution (A)
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
12,000 mg · oral · single doseAs given in this study (human).
Source sentences
Summary
All other treatment comparisons (C vs A and B vs C) also met the bioequivalence criteria.
2 context sentences(design, population, supporting numbers)
Summary · context
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_max), area under the concentration-time curve from time 0 to time t (AUC_0-t), and AUC from time 0 to in…
Summary · context
In 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; treatmen…
Report a problemFND-4167813704
05HumanNon-randomized trialPharmacokinetics
In healthy adults, trofinetide powder in 25 mL water met AUC0-∞ bioequivalence criteria versus ready-to-use solution.
In plain wordsIn healthy adults, trofinetide powder in 25 mL water and ready-to-use solution had equivalent total exposure to infinity.
Peptide
Trofinetide
Outcome
No significant changeAUC0-infinity bioequivalence: C versus A
Study
n = 38
Comparator
ready-to-use oral solution (A)
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
12,000 mg · oral · single doseAs given in this study (human).
Source sentences
Summary
All other treatment comparisons (C vs A and B vs C) also met the bioequivalence criteria.
2 context sentences(design, population, supporting numbers)
Summary · context
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_max), area under the concentration-time curve from time 0 to time t (AUC_0-t), and AUC from time 0 to in…
Summary · context
In 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; treatmen…
Report a problemFND-4167813705
06HumanNon-randomized trialPharmacokinetics
In healthy adults, trofinetide powder in 25 mL water met Cmax bioequivalence criteria versus ready-to-use solution.
In plain wordsIn healthy adults, trofinetide powder in 25 mL water and ready-to-use solution had equivalent peak concentrations.
Peptide
Trofinetide
Outcome
No significant changeCmax bioequivalence: C versus A
Study
n = 38
Comparator
ready-to-use oral solution (A)
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
12,000 mg · oral · single doseAs given in this study (human).
Source sentences
Summary
All other treatment comparisons (C vs A and B vs C) also met the bioequivalence criteria.
2 context sentences(design, population, supporting numbers)
Summary · context
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_max), area under the concentration-time curve from time 0 to time t (AUC_0-t), and AUC from time 0 to in…
Summary · context
In 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; treatmen…
Report a problemFND-4167813706
07HumanNon-randomized trialPharmacokinetics
In healthy adults, trofinetide powder in 60 mL water met AUC0-t bioequivalence criteria versus powder in 25 mL water.
In plain wordsIn healthy adults, trofinetide powder in 60 mL water and powder in 25 mL water had equivalent AUC0-t.
Peptide
Trofinetide
Outcome
No significant changeAUC0-t bioequivalence: B versus C
Study
n = 38
Comparator
powder constituted in 25 mL water (C)
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
12,000 mg · oral · single doseAs given in this study (human).
Source sentences
Summary
All other treatment comparisons (C vs A and B vs C) also met the bioequivalence criteria.
2 context sentences(design, population, supporting numbers)
Summary · context
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_max), area under the concentration-time curve from time 0 to time t (AUC_0-t), and AUC from time 0 to in…
Summary · context
In 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; treatmen…
Report a problemFND-4167813707
08HumanNon-randomized trialPharmacokinetics
In healthy adults, trofinetide powder in 60 mL water met AUC0-∞ bioequivalence criteria versus powder in 25 mL water.
In plain wordsIn healthy adults, trofinetide powder in 60 mL water and powder in 25 mL water had equivalent total exposure to infinity.
Peptide
Trofinetide
Outcome
No significant changeAUC0-infinity bioequivalence: B versus C
Study
n = 38
Comparator
powder constituted in 25 mL water (C)
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
12,000 mg · oral · single doseAs given in this study (human).
Source sentences
Summary
All other treatment comparisons (C vs A and B vs C) also met the bioequivalence criteria.
2 context sentences(design, population, supporting numbers)
Summary · context
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_max), area under the concentration-time curve from time 0 to time t (AUC_0-t), and AUC from time 0 to in…
Summary · context
In 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; treatmen…
Report a problemFND-4167813708
09HumanNon-randomized trialPharmacokinetics
In healthy adults, trofinetide powder in 60 mL water met Cmax bioequivalence criteria versus powder in 25 mL water.
In plain wordsIn healthy adults, trofinetide powder in 60 mL water and powder in 25 mL water had equivalent peak concentrations.
Peptide
Trofinetide
Outcome
No significant changeCmax bioequivalence: B versus C
Study
n = 38
Comparator
powder constituted in 25 mL water (C)
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
12,000 mg · oral · single doseAs given in this study (human).
Source sentences
Summary
All other treatment comparisons (C vs A and B vs C) also met the bioequivalence criteria.
2 context sentences(design, population, supporting numbers)
Summary · context
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_max), area under the concentration-time curve from time 0 to time t (AUC_0-t), and AUC from time 0 to in…
Summary · context
In 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; treatmen…
Report a problemFND-4167813709
10HumanNon-randomized trialSafety outcome
In healthy adults receiving trofinetide formulations, diarrhea was the most frequent treatment-emergent adverse event (6 participants, 15.8%).
In plain wordsIn healthy adults receiving trofinetide formulations, diarrhea was the most frequent adverse event, affecting 6 participants (15.8%).
Peptide
Trofinetide
Outcome
Not applicableDiarrhea
Study
n = 38
Outcome group
Gastrointestinal adverse events · Gastrointestinal
Studied dose
12,000 mg · oral · single doseAs given in this study (human).
Measurements
Diarrhea
6 participants
all formulations
Diarrhea
15.8%
all formulations
Source sentences
Summary
The most frequent treatment-emergent adverse event was diarrhea (n = 6 [15.8%]).
1 context sentence(design, population, supporting numbers)
Summary · context
In 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; treatmen…
Report a problemFND-4167813710
11HumanNon-randomized trialSafety outcome
In healthy adults receiving trofinetide formulations, no serious adverse events occurred.
In plain wordsIn healthy adults receiving trofinetide formulations, no serious adverse events occurred.
Peptide
Trofinetide
Outcome
Not applicableSerious adverse events
Study
n = 38
Outcome group
Adverse events · Whole body
Studied dose
12,000 mg · oral · single doseAs given in this study (human).
Source sentences
Summary
No serious or unexpected adverse events occurred during the study.
1 context sentence(design, population, supporting numbers)
Summary · context
In 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; treatmen…
Report a problemFND-4167813711
12HumanNon-randomized trialSafety outcome
In healthy adults receiving trofinetide formulations, no unexpected adverse events occurred.
In plain wordsIn healthy adults receiving trofinetide formulations, no unexpected adverse events occurred.
Peptide
Trofinetide
Outcome
Not applicableUnexpected adverse events
Study
n = 38
Outcome group
Adverse events · Whole body
Studied dose
12,000 mg · oral · single doseAs given in this study (human).
Source sentences
Summary
No serious or unexpected adverse events occurred during the study.
1 context sentence(design, population, supporting numbers)
Summary · context
In 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; treatmen…
Report a problemFND-4167813712

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
28 Oct 2026, 02:03 UTC
Study results updated
Version 1
27 Oct 2026, 16:37 UTC
First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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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.

Summary

Results from this study12

Version history

Subjects

Publication types

MeSH terms

Keywords