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Study · Randomized controlled trial · n = 121 · 2020

2 results

SAT-LB18 A Randomized Controlled Trial of Vosoritide in Children With Achondroplasia

Savarirayan R, Tofts L, Irving M, Wilcox W, Bacino C, Hoover-Fong J, Font R, Harmatz P, Rutsch F, Bober M, Polgreen L, Ginebreda I and 19 more

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Mohnike K, Charrow J, Hoernschmeyer D, Ozono K, Alanay Y, Arundel P, Kagami S, Yasui N, White K, Saal H, Leiva-Gea A, Luna-Gonzáles F, Mochizuki H, Basel D, Porco D, Jayaram K, Fisheleva E, Huntsman-Labed A, Day J

Journal of the Endocrine Society8 May 2020

PMCIDPMC7209563 (opens in a new tab)PepBankPUB-97194349
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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 study2

2 results from studies that gave the peptide. 2 results from people.

  1. 01HumanRandomized controlled trialEfficacy outcome

    In children with achondroplasia, vosoritide increased annualized growth velocity by 1.57 cm/year versus placebo (95% CI 1.22, 1.93; p<0.001).

    In plain wordsIn children with achondroplasia, vosoritide increased annualized growth velocity by 1.57 cm/year compared with placebo.

    Peptide
    Vosoritide
    Outcome
    IncreaseAnnualized growth velocity change
    Study

    n = 121

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 μg/kg once daily · subcutaneous · 52 weeksAs given in this study (human).

    Measurements

    Adjusted mean difference in annualized growth velocity
    1.57 cm/year
    vosoritide versus placebo
    95% CI
    1.22 to 1.93 cm/year
    adjusted mean difference
    p-value
    < 0.001
    vosoritide versus placebo

    Source sentences

    Summary
    The adjusted mean difference in annualized growth velocity between patients administered vosoritide and those administered placebo was 1.57 cm per year in favor of vosoritide (95% CI: [1.22, 1.93], two-sided p-value <0.001).
    1 context sentence
    Summary · context
    This international, randomized, double-blind, phase 3 trial compared once-daily subcutaneous administration of vosoritide, at a dose of 15 μg per kg of body weight, with placebo in children with achondroplasia aged 5 to <18 years.
  2. 02HumanRandomized controlled trialSafety outcome

    In children with achondroplasia, the incidence of adverse events was similar with vosoritide (59 patients, 98.3%) and placebo (60 patients, 98.4%).

    In plain wordsIn children with achondroplasia, adverse events were similar with vosoritide (98.3%) and with placebo (98.4%).

    Peptide
    Vosoritide
    Outcome
    DecreaseAny adverse event
    Study

    n = 121

    Comparator
    Placebo
    Outcome group
    Adverse events · Whole body
    Studied dose
    15 μg/kg once daily · subcutaneous · 52 weeksAs given in this study (human).

    Measurements

    Patients with at least one adverse event
    59 patients
    vosoritide
    Proportion with adverse event
    98.3%
    vosoritide
    Patients with at least one adverse event
    60 patients
    placebo
    Proportion with adverse event
    98.4%
    placebo

    Source sentences

    Summary
    A total of 119 patients experienced at least one adverse event (vosoritide group, 59 [98.3%], placebo group, 60 [98.4%]).
    1 context sentence
    Summary · context
    This international, randomized, double-blind, phase 3 trial compared once-daily subcutaneous administration of vosoritide, at a dose of 15 μg per kg of body weight, with placebo in children with achondroplasia aged 5 to <18 years.

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.

  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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