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Study · Randomized controlled trial · 2020

4 results

A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Fragile X Syndrome.

Berry-Kravis E, Horrigan JP, Tartaglia N, Hagerman R, Kolevzon A, Erickson CA, Hatti S, Snape M, Yaroshinsky A, Stoms G, Glass L, Jones NE

Pediatric neurology23 May 2020Cited by 35 (Europe PMC)

PMID32660869 (opens in a new tab)DOI10.1016/j.pediatrneurol.2020.04.019 (opens in a new tab)PepBankPUB-32660869
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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-ND. Results still quote short attributed sentences.

Results from this study4

4 results from studies that gave the peptide. 4 results from people.

  1. 01HumanRandomized controlled trialEfficacy outcome

    In males with fragile X syndrome, trofinetide at 70 mg/kg met prespecified efficacy criteria versus placebo, with false-positive probability 0.045.

    In plain wordsIn males with fragile X syndrome, trofinetide at 70 mg/kg met prespecified benefit criteria versus placebo.

    Peptide
    Trofinetide
    Outcome
    Not applicablePrespecified efficacy criteria
    Study

    n: not reported in source

    Comparator
    Placebo
    Outcome group
    Fragile X Syndrome Symptoms · Nervous system and cognition
    Studied dose
    70 mg/kg twice daily · oral · 28 daysAs given in this study (human).

    Measurements

    False-positive probability
    0.045
    permutation test

    Source sentences

    Summary
    Trofinetide at the 70 mg/kg dose level demonstrated efficacy compared with placebo based on prespecified criteria.
    3 context sentences
    Summary · context
    On the basis of a permutation test, the probability of a false-positive outcome for the achieved prespecified success was 0.045.
    Summary · context
    This study was an exploratory, phase 2, multicenter, double-blind, placebo-controlled, parallel group study of the safety and tolerability of orally administered trofinetide in 72 adolescent and adult males with fragile X syndrome.
    Summary · context
    Subjects were randomly assigned in a 1:1:1 ratio to 35 or 70 mg/kg twice daily trofinetide or placebo for 28 days.
  2. 02HumanNon-randomized trialSafety outcome

    In males with fragile X syndrome, trofinetide at 35 mg/kg twice daily was well tolerated and appeared generally safe.

    In plain wordsIn males with fragile X syndrome, trofinetide at 35 mg/kg twice daily was well tolerated and appeared generally safe.

    Peptide
    Trofinetide
    Outcome
    Not applicableSafety and tolerability at 35 mg/kg
    Study

    n: not reported in source

    Outcome group
    Adverse events · Whole body
    Studied dose
    35 mg/kg twice daily · oral · 28 daysAs given in this study (human).

    Source sentences

    Summary
    Both 35 and 70 mg/kg dose levels of trofinetide were well tolerated and appeared to be generally safe.
    2 context sentences
    Summary · context
    This study was an exploratory, phase 2, multicenter, double-blind, placebo-controlled, parallel group study of the safety and tolerability of orally administered trofinetide in 72 adolescent and adult males with fragile X syndrome.
    Summary · context
    Subjects were randomly assigned in a 1:1:1 ratio to 35 or 70 mg/kg twice daily trofinetide or placebo for 28 days.
  3. 03HumanNon-randomized trialSafety outcome

    In males with fragile X syndrome, trofinetide at 70 mg/kg twice daily was well tolerated and appeared generally safe.

    In plain wordsIn males with fragile X syndrome, trofinetide at 70 mg/kg twice daily was well tolerated and appeared generally safe.

    Peptide
    Trofinetide
    Outcome
    Not applicableSafety and tolerability at 70 mg/kg
    Study

    n: not reported in source

    Outcome group
    Adverse events · Whole body
    Studied dose
    70 mg/kg twice daily · oral · 28 daysAs given in this study (human).

    Source sentences

    Summary
    Both 35 and 70 mg/kg dose levels of trofinetide were well tolerated and appeared to be generally safe.
    2 context sentences
    Summary · context
    This study was an exploratory, phase 2, multicenter, double-blind, placebo-controlled, parallel group study of the safety and tolerability of orally administered trofinetide in 72 adolescent and adult males with fragile X syndrome.
    Summary · context
    Subjects were randomly assigned in a 1:1:1 ratio to 35 or 70 mg/kg twice daily trofinetide or placebo for 28 days.
  4. 04HumanNon-randomized trialEfficacy outcome

    In males with fragile X syndrome in this trial of trofinetide, a group analysis showed improvement from treatment baseline on three unnamed fragile X-specific outcome measures; the abstract does not say which arms were analysed.

    In plain wordsIn a fragile X trial, a group analysis showed improvement from treatment baseline on three unnamed measures, without naming the arms.

    Peptide
    Trofinetide
    Outcome
    Not applicableThree fragile X-specific outcome measures
    Study

    n: not reported in source

    Comparator
    Treatment baseline
    Outcome group
    Fragile X Syndrome Symptoms · Nervous system and cognition
    Studied dose
    Dose not stated · oral · 28 daysAs given in this study (human).

    Source sentences

    Summary
    In the group analysis, improvement from treatment baseline was demonstrated on three fragile X syndrome-specific outcome measures.
    2 context sentences
    Summary · context
    This study was an exploratory, phase 2, multicenter, double-blind, placebo-controlled, parallel group study of the safety and tolerability of orally administered trofinetide in 72 adolescent and adult males with fragile X syndrome.
    Summary · context
    Subjects were randomly assigned in a 1:1:1 ratio to 35 or 70 mg/kg twice daily trofinetide or placebo for 28 days.

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

Registered trials1

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

1 trial · 1 completed · source ClinicalTrials.gov (opens in a new tab)

CompletedPhase 2NCT01894958

A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome

Neuren Pharmaceuticals Limitedn = 72Jan 2014 to Oct 2015

Fragile X Syndrome

Trial pageClinicalTrials.govUpdated 5 Feb 2018

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.

  1. Version 2Current

    28 Oct 2026, 02:03 UTC

    Study results updated

  2. 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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