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Study · Non-randomized trial · n = 75 · 2022

7 results

LBMON196 A Randomized Controlled Trial Of Vosoritide In Infants And Toddlers With Achondroplasia

The source lists no authors.

Journal of the Endocrine Society1 Nov 2022

PMCIDPMC9625654 (opens in a new tab)PepBankPUB-91664474
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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 study7

7 results from studies that gave the peptide. 7 results from people.

  1. 01HumanNon-randomized trialSafety outcome

    In young children with achondroplasia, all enrolled patients, including those given vosoritide and those given placebo, reported at least one adverse event.

    In plain wordsIn young children with achondroplasia, every patient reported at least one adverse event, including patients given placebo.

    Peptide
    Vosoritide
    Outcome
    Not applicableAny adverse event
    Study

    n = 75

    Analysis
    Primary
    Outcome group
    Adverse events · Whole body
    Studied dose
    15 or 30 μg/kg once daily · subcutaneous · 52 weeksAs given in this study (human).

    Source sentences

    Summary
    All patients reported at least one adverse event.
    2 context sentences
    Summary · context
    This global, phase 2, randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of vosoritide on growth in children with achondroplasia aged 3 months to <5 years.
    Summary · context
    A further 32 were randomized to receive vosoritide and 32 to receive placebo.
  2. 02HumanNon-randomized trialEfficacy outcome

    In young children with achondroplasia, vosoritide increased height Z-score by 0.30 SD versus placebo at week 52 (95% CI zero point zero seven, 0.54).

    In plain wordsIn young children with achondroplasia, vosoritide increased height Z-score by 0.30 SD versus placebo at week 52.

    Peptide
    Vosoritide
    Outcome
    IncreaseHeight Z-score change at week 52
    Study

    n = 75

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 or 30 μg/kg once daily · subcutaneous · 52 weeksAs given in this study (human).

    Measurements

    Height Z-score difference versus placebo
    0.3 SD
    Week 52, full analysis population (n=43 vosoritide, n=32 placebo)

    Source sentences

    Summary
    In the full analysis population, vosoritide (n=43) compared to placebo (n=32), increased height Z-score by 0.30 SD (95% CI 0. 07, 0.54);
    2 context sentences
    Summary · context
    This global, phase 2, randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of vosoritide on growth in children with achondroplasia aged 3 months to <5 years.
    Summary · context
    A further 32 were randomized to receive vosoritide and 32 to receive placebo.
  3. 03HumanNon-randomized trialEfficacy outcome

    In young children with achondroplasia, vosoritide increased annualized growth velocity by 0.92 cm/year versus placebo (95% CI 0.24, 1.59).

    In plain wordsIn young children with achondroplasia, vosoritide increased annualized growth velocity by 0.92 cm/year versus placebo.

    Peptide
    Vosoritide
    Outcome
    IncreaseAnnualized growth velocity change at week 52
    Study

    n = 75

    Comparator
    Placebo
    Analysis
    Secondary
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 or 30 μg/kg once daily · subcutaneous · 52 weeksAs given in this study (human).

    Measurements

    Annualized growth velocity difference versus placebo
    0.92 cm/year
    Week 52, full analysis population (n=43 vosoritide, n=32 placebo)
    95% CI of annualized growth velocity difference
    0.24 to 1.59 cm/year
    Week 52

    Source sentences

    Summary
    increased AGV by 0.92cm/year (95% CI 0.24, 1.59);
    2 context sentences
    Summary · context
    In the full analysis population, vosoritide (n=43) compared to placebo (n=32),
    Summary · context
    This global, phase 2, randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of vosoritide on growth in children with achondroplasia aged 3 months to <5 years.
  4. 04HumanNon-randomized trialEfficacy outcome

    In young children with achondroplasia, vosoritide did not worsen upper-to-lower body segment ratio, which changed by minus zero point zero six versus placebo (95% CI -0.15, zero point zero three).

    In plain wordsIn young children with achondroplasia, vosoritide did not worsen body segment ratio, which changed by minus zero point zero six versus placebo.

    Peptide
    Vosoritide
    Outcome
    DecreaseUpper-to-lower body segment ratio change at week 52
    Study

    n = 75

    Comparator
    Placebo
    Analysis
    Secondary
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 or 30 μg/kg once daily · subcutaneous · 52 weeksAs given in this study (human).

    Measurements

    95% CI of ratio change
    −0.15 to 0.03 ratio
    Week 52

    Source sentences

    Summary
    and did not worsen upper-to-lower body segment ratio which changed by -0. 06 (95% CI -0.15, 0. 03).
    2 context sentences
    Summary · context
    In the full analysis population, vosoritide (n=43) compared to placebo (n=32),
    Summary · context
    This global, phase 2, randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of vosoritide on growth in children with achondroplasia aged 3 months to <5 years.
  5. 05HumanNon-randomized trialSafety outcome

    Children receiving vosoritide had four serious adverse events versus eight with placebo, none treatment-related.

    In plain wordsChildren receiving vosoritide had four serious adverse events versus eight with placebo, none treatment-related.

    Peptide
    Vosoritide
    Outcome
    DecreaseSerious adverse events
    Study

    n: not reported in source

    Comparator
    Placebo
    Outcome group
    Adverse events · Whole body
    Studied dose
    15 or 30 μg/kg once daily · subcutaneous · 52 weeksAs given in this study (human).

    Measurements

    Serious adverse events
    4 events
    Vosoritide
    Serious adverse events
    8 events
    Placebo

    Source sentences

    Summary
    Four serious adverse events occurred with vosoritide and 8 with placebo, none were treatment-related.
    2 context sentences
    Summary · context
    This global, phase 2, randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of vosoritide on growth in children with achondroplasia aged 3 months to <5 years.
    Summary · context
    A further 32 were randomized to receive vosoritide and 32 to receive placebo.
  6. 06HumanNon-randomized trialSafety outcome

    One child receiving vosoritide discontinued after a fatal respiratory arrest and had pre-existing respiratory morbidity.

    In plain wordsOne child receiving vosoritide discontinued after a fatal respiratory arrest and had pre-existing respiratory morbidity.

    Peptide
    Vosoritide
    Outcome
    Not applicableDiscontinuation
    Study

    n: not reported in source

    Outcome group
    Adverse events · Whole body
    Studied dose
    15 or 30 μg/kg once daily · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Two participants discontinued, one on vosoritide with pre-existing respiratory morbidity who had a fatal respiratory arrest and one on placebo who withdrew consent.
    2 context sentences
    Summary · context
    This global, phase 2, randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of vosoritide on growth in children with achondroplasia aged 3 months to <5 years.
    Summary · context
    A further 32 were randomized to receive vosoritide and 32 to receive placebo.
  7. 07HumanNon-randomized trialSafety outcome

    One child receiving vosoritide with pre-existing respiratory morbidity had a fatal respiratory arrest.

    In plain wordsOne child receiving vosoritide with pre-existing respiratory morbidity had a fatal respiratory arrest.

    Peptide
    Vosoritide
    Outcome
    Not applicableFatal respiratory arrest
    Study

    n: not reported in source

    Outcome group
    Mortality · Respiratory
    Studied dose
    15 or 30 μg/kg once daily · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Two participants discontinued, one on vosoritide with pre-existing respiratory morbidity who had a fatal respiratory arrest and one on placebo who withdrew consent.
    2 context sentences
    Summary · context
    This global, phase 2, randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of vosoritide on growth in children with achondroplasia aged 3 months to <5 years.
    Summary · context
    A further 32 were randomized to receive vosoritide and 32 to receive placebo.

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.

Publication types

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