Peptide LibraryResearch statistics
Trofinetide research: publications and trials
At a glance
Publications, all years
67
Since 2009
Publications in 2026 so far
14
Through November 1, 2026
Human clinical research
47.5%
28 of 59 classified
Registered clinical trials
13
ClinicalTrials.gov
Randomized controlled trials
9
Publications reporting an RCT in people
Systematic reviews and meta-analyses
3
Preclinical studies
20.3%
8 animal · 0 in vitro · 4 computational
Records that only mention it
20
Not counted above
Status in the library: FDA-approved. 8 publications are not yet classified by study type.
What to keep in mind
- Some completed trials have neither posted results nor a linked results publication.
Publications by year
Trofinetide publications by year and evidence
Show the data
| Year | Human clinical research | Preclinical (animal, in vitro, computational) | Reviews and meta-analyses | Other and not yet classified | Total |
|---|---|---|---|---|---|
| 2009 | 0 | 4 | 0 | 0 | 4 |
| 2013 | 0 | 1 | 0 | 0 | 1 |
| 2015 | 0 | 1 | 0 | 0 | 1 |
| 2017 | 2 | 0 | 0 | 0 | 2 |
| 2019 | 1 | 0 | 0 | 0 | 1 |
| 2020 | 1 | 0 | 0 | 0 | 1 |
| 2022 | 1 | 0 | 0 | 1 | 2 |
| 2023 | 3 | 0 | 4 | 4 | 11 |
| 2024 | 6 | 3 | 6 | 5 | 20 |
| 2025 | 5 | 1 | 2 | 2 | 10 |
| 2026 (to date) | 9 | 2 | 1 | 2 | 14 |
Study types
| Observational human study | 10 |
|---|---|
| Review | 10 |
| Randomized controlled trial | 9 |
| Animal study | 8 |
| Not yet classified | 8 |
| Other clinical trial | 7 |
| Editorial, letter or other | 5 |
| Computational study | 4 |
| Meta-analysis | 3 |
| Case report | 2 |
| Guideline or consensus | 1 |
Research areas
From MeSH major topics. A paper can be in several areas.
| Neurological and mental health | 56 |
|---|---|
| Cognitive health | 4 |
| Gastrointestinal | 4 |
| Tissue repair | 3 |
| Cardiovascular | 2 |
| Immune system | 1 |
| Longevity and aging | 1 |
| Reproductive and sexual health | 1 |
Registered clinical trials
3 of 9 trials completed at least a year before the cutoff have neither posted results nor a linked results publication. A registration is not a result.
Completed: 9 · Terminated: 3 · Not yet recruiting: 1
Phase not applicable: 1 · Phase 1: 2 · Phase 2: 6 · Phase 2 / Phase 3: 1 · Phase 3: 3
Human trials and evidence reviews
Meta-analyses, systematic reviews and trials in people, newest first.
- Is trofinetide a future treatment for Rett syndrome? A comprehensive systematic review and meta-analysis of randomized controlled trials.
BMC medicine · July 18, 2024 · Meta-analysis · cited 3 times · open access
PMID 39020317 · DOI 10.1186/s12916-024-03506-9 · Reviews and meta-analyses
- A meta-analysis of the efficacy and safety of trofinetide in patients with rett syndrome.
Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · May 21, 2024 · Meta-analysis · cited 5 times · open access
PMID 38771525 · DOI 10.1007/s10072-024-07584-8 · Reviews and meta-analyses
- Safety and efficacy of trofinetide in Rett syndrome: a systematic review and meta-analysis of randomized controlled trials.
BMC pediatrics · March 23, 2024 · Meta-analysis · cited 8 times · open access
PMID 38521908 · DOI 10.1186/s12887-024-04526-3 · Reviews and meta-analyses
- 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 · February 12, 2026 · Randomized controlled trial · cited 0 times · open access
PMID 41678137 · DOI 10.1007/s12325-025-03484-1 · Human clinical research
- Trofinetide for the treatment of Rett syndrome: Results from the open-label extension LILAC study.
Med (New York, N.Y.) · June 24, 2024 · Randomized controlled trial · cited 39 times · open access
PMID 38917793 · DOI 10.1016/j.medj.2024.05.018 · Human clinical research
- Trofinetide Treatment Demonstrates a Benefit Over Placebo for the Ability to Communicate in Rett Syndrome.
Pediatric neurology · November 23, 2023 · Randomized controlled trial · cited 25 times · open access
PMID 38232652 · DOI 10.1016/j.pediatrneurol.2023.11.005 · Human clinical research
- Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study.
Nature medicine · June 8, 2023 · Randomized controlled trial · cited 195 times · open access
PMID 37291210 · DOI 10.1038/s41591-023-02398-1 · Human clinical research
- 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.
Clinical drug investigation · May 27, 2022 · Randomized controlled trial · cited 21 times · open access
PMID 35622206 · DOI 10.1007/s40261-022-01156-4 · Human clinical research
- A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Fragile X Syndrome.
Pediatric neurology · May 23, 2020 · Randomized controlled trial · cited 102 times · open access
PMID 32660869 · DOI 10.1016/j.pediatrneurol.2020.04.019 · Human clinical research
- Double-blind, randomized, placebo-controlled study of trofinetide in pediatric Rett syndrome.
Neurology · March 27, 2019 · Randomized controlled trial · cited 160 times · open access
PMID 30918097 · DOI 10.1212/wnl.0000000000007316 · Human clinical research
Most cited
By OpenAlex citation count. Citations measure attention, not quality or correctness.
- Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study.
Nature medicine · June 8, 2023 · Randomized controlled trial · cited 195 times · open access
PMID 37291210 · DOI 10.1038/s41591-023-02398-1 · Human clinical research
- Double-blind, randomized, placebo-controlled study of trofinetide in pediatric Rett syndrome.
Neurology · March 27, 2019 · Randomized controlled trial · cited 160 times · open access
PMID 30918097 · DOI 10.1212/wnl.0000000000007316 · Human clinical research
- A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Rett Syndrome.
Pediatric neurology · July 8, 2017 · Randomized controlled trial · cited 104 times
PMID 28964591 · DOI 10.1016/j.pediatrneurol.2017.07.002 · Human clinical research
- A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Fragile X Syndrome.
Pediatric neurology · May 23, 2020 · Randomized controlled trial · cited 102 times · open access
PMID 32660869 · DOI 10.1016/j.pediatrneurol.2020.04.019 · Human clinical research
- NNZ-2566 treatment inhibits neuroinflammation and pro-inflammatory cytokine expression induced by experimental penetrating ballistic-like brain injury in rats.
Journal of neuroinflammation · August 5, 2009 · Animal study · cited 81 times · open access
PMID 19656406 · DOI 10.1186/1742-2094-6-19 · Animal studies
Latest publications
- Remarkable seizure reduction with trofinetide treatment in an adult with Rett syndrome: A case report.
Seizure · August 18, 2026 · Case report · cited 0 times · open access
PMID 42679656 · DOI 10.1016/j.seizure.2026.08.016 · Human clinical research
- Mapping Trofinetide Polypharmacology in Rett Syndrome: A Multi-Stage Computational Analysis.
Journal of computational chemistry · August 1, 2026 · Computational study · cited 0 times · open access
PMID 42530159 · DOI 10.1002/jcc.70468 · Computational studies
- Characteristics of adult individuals with Rett syndrome treated or untreated with trofinetide in the United States.
Frontiers in neurology · July 27, 2026 · Observational human study · cited 1 times · open access
PMID 42577214 · DOI 10.3389/fneur.2026.1856312 · Human clinical research
- Real-World Demographics, Clinical Characteristics, and Predictors of Persistence Among Males with Rett Syndrome Treated with Trofinetide.
Advances in therapy · June 30, 2026 · Observational human study · cited 0 times · open access
PMID 42377739 · DOI 10.1007/s12325-026-03686-1 · Human clinical research
- Trofinetide Use and Treatment Patterns Among Children Aged 2-4 Years with Rett Syndrome in the United States: A Retrospective Specialty Pharmacy Linked Claims Database Analysis.
International journal of general medicine · June 29, 2026 · Observational human study · cited 0 times · open access
PMID 42404293 · DOI 10.2147/ijgm.s627959 · Human clinical research
- Rett syndrome and real-world treatment patterns of trofinetide in the United States.
Journal of medical economics · June 13, 2026 · Observational human study · cited 2 times · open access
PMID 42287113 · DOI 10.1080/13696998.2026.2682668 · Human clinical research
- Real-World Treatment Landscape of Individuals with Rett Syndrome Treated and Untreated with Trofinetide in the United States.
Journal of health economics and outcomes research · June 11, 2026 · Observational human study · cited 1 times · open access
PMID 42294489 · DOI 10.36469/001c.162589 · Human clinical research
- Clinical experience using trofinetide in Rett syndrome and related MECP2 diagnosis at the children's hospital of Philadelphia post approval.
Frontiers in neurology · April 23, 2026 · Observational human study · cited 0 times · open access
PMID 42111068 · DOI 10.3389/fneur.2026.1776146 · Human clinical research
- Design, synthesis and evaluation of trofinetide prodrug based on diketopiperazine strategy.
Bioorganic & medicinal chemistry · March 14, 2026 · Animal study · cited 1 times
PMID 41850142 · DOI 10.1016/j.bmc.2026.118633 · Animal studies
- Expert Consensus on Real-World Use of Trofinetide for Rett Syndrome Using a Modified Delphi Method.
Annals of the Child Neurology Society · March 6, 2026 · Guideline or consensus · cited 7 times · open access
PMID 42563928 · DOI 10.1002/cns3.70062 · Commentary and other
How this is counted
PubMed records matched by the names below, counted when the peptide is in the title, indexed by MeSH, or confirmed from the abstract. The exact query can be rerun on Europe PMC. Methodology
Search terms: ACP-2566; Daybue; Daybue Stix; NNZ-2566; Trofinetide
((TITLE_ABS:"ACP-2566" OR CHEM:"ACP-2566" OR TITLE_ABS:"ACP2566" OR CHEM:"ACP2566") OR (TITLE_ABS:"Daybue" OR CHEM:"Daybue") OR (TITLE_ABS:"Daybue Stix" OR CHEM:"Daybue Stix") OR (TITLE_ABS:"NNZ-2566" OR CHEM:"NNZ-2566" OR TITLE_ABS:"NNZ2566" OR CHEM:"NNZ2566") OR (TITLE_ABS:"Trofinetide" OR CHEM:"Trofinetide")) AND SRC:MED
Cite this page
Peptide Library. (2026, November 2). Trofinetide research statistics (Version weekly-2026-11-01) [Statistic]. Peptide Library. https://peptidelibrary.app/research/peptides/trofinetide