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Study · Randomized controlled trial · n = 75 · 2024

9 results

Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial.

Savarirayan R, Wilcox WR, Harmatz P, Phillips J 3rd, Polgreen LE, Tofts L, Ozono K, Arundel P, Irving M, Bacino CA, Basel D, Bober MB and 11 more

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Charrow J, Mochizuki H, Kotani Y, Saal HM, Army C, Jeha G, Qi Y, Han L, Fisheleva E, Huntsman-Labed A, Day J

The Lancet. Child & adolescent health18 Nov 2023Cited by 59 (Europe PMC)

PMID37984383 (opens in a new tab)DOI10.1016/s2352-4642(23)00265-1 (opens in a new tab)PepBankPUB-37984383
Publisher pagePubMedAsk about this study
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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: Publisher copyright. Results still quote short attributed sentences.

Results from this study9

9 results from studies that gave the peptide. 9 results from people.

  1. 01HumanRandomized controlled trialEfficacy outcome

    In young children with achondroplasia, vosoritide exceeded placebo in height Z-score change by 0.25 (95% CI -0.02 to 0.53).

    In plain wordsIn young children with achondroplasia, height score change favored vosoritide over placebo, but the confidence interval included zero.

    Peptide
    Vosoritide
    Outcome
    IncreaseHeight Z-score change
    Study

    n = 64

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    30.0 μg/kg for ages 0-23 months; 15.0 μg/kg for ages 24-59 months · subcutaneous · 52 weeksAs given in this study (human).

    Measurements

    Least-squares mean difference in height Z score change from baseline
    0.25 Z score
    Vosoritide vs placebo
    95% CI of mean difference
    −0.02 to 0.53 Z score
    Vosoritide vs placebo

    Source sentences

    Summary
    The least-squares mean difference for change from baseline in height Z score between the vosoritide and placebo groups was 0·25 (95% CI -0·02 to 0·53).
    3 context sentences
    Summary · context
    The second primary outcome was change in height Z score at 52 weeks from baseline, analysed in all randomly assigned participants.
    Summary · context
    11 were assigned as sentinels, whereas 32 were randomly assigned to receive vosoritide and 32 placebo.
    Summary · context
    This double-blind, randomised, placebo-controlled, phase 2 trial was done in 16 hospitals across Australia, Japan, the UK, and the USA.
  2. 02HumanNon-randomized trialSafety outcome

    In young children with achondroplasia, all vosoritide recipients experienced adverse events, as did all study participants.

    In plain wordsAll young children receiving vosoritide experienced adverse events, as did all study participants.

    Peptide
    Vosoritide
    Outcome
    Not applicableAdverse event occurrence
    Study

    n = 75

    Analysis
    Primary
    Outcome group
    Adverse events · Whole body
    Studied dose
    30.0 μg/kg for ages 0-23 months; 15.0 μg/kg for ages 24-59 months · subcutaneous · 52 weeksAs given in this study (human).

    Measurements

    Participants with adverse events
    100%
    All 75 participants, vosoritide and placebo

    Source sentences

    Summary
    Adverse events occurred in all 75 (100%) participants (annual rate 204·5 adverse events per patient in the vosoritide group and 73·6 per patient in the placebo group), most of which were transient injection-site reactions and injection-site erythema.
    1 context sentence
    Summary · context
    This double-blind, randomised, placebo-controlled, phase 2 trial was done in 16 hospitals across Australia, Japan, the UK, and the USA.
  3. 03HumanNon-randomized trialSafety outcome

    In young children with achondroplasia, annual adverse event rates were 204.5 per vosoritide patient versus 73.6 per placebo patient.

    In plain wordsIn young children with achondroplasia, adverse event rates were higher with vosoritide than placebo.

    Peptide
    Vosoritide
    Outcome
    IncreaseAnnual adverse event rate
    Study

    n = 75

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Adverse events · Whole body
    Studied dose
    30.0 μg/kg for ages 0-23 months; 15.0 μg/kg for ages 24-59 months · subcutaneous · 52 weeksAs given in this study (human).

    Measurements

    Annual adverse event rate
    204.5 adverse events per patient per year
    Vosoritide group
    Annual adverse event rate
    73.6 adverse events per patient per year
    Placebo group

    Source sentences

    Summary
    Adverse events occurred in all 75 (100%) participants (annual rate 204·5 adverse events per patient in the vosoritide group and 73·6 per patient in the placebo group), most of which were transient injection-site reactions and injection-site erythema.
    1 context sentence
    Summary · context
    This double-blind, randomised, placebo-controlled, phase 2 trial was done in 16 hospitals across Australia, Japan, the UK, and the USA.
  4. 04HumanNon-randomized trialSafety outcome

    In young children with achondroplasia receiving vosoritide, one participant died and discontinued treatment and the study.

    In plain wordsOne young child receiving vosoritide died and discontinued the study.

    Peptide
    Vosoritide
    Outcome
    Not applicableDeath
    Study

    n: not reported in source

    Analysis
    Primary
    Outcome group
    Mortality · Whole body
    Studied dose
    30.0 μg/kg for ages 0-23 months; 15.0 μg/kg for ages 24-59 months · subcutaneous · 52 weeksAs given in this study (human).

    Source sentences

    Summary
    Two participants discontinued treatment and the study: one in the vosoritide group (death) and one in the placebo group (withdrawal).
    1 context sentence
    Summary · context
    This double-blind, randomised, placebo-controlled, phase 2 trial was done in 16 hospitals across Australia, Japan, the UK, and the USA.
  5. 05HumanNon-randomized trialSafety outcome

    In young children with achondroplasia, serious adverse events occurred in three (7%) vosoritide recipients versus six (19%) placebo recipients.

    In plain wordsIn young children with achondroplasia, serious adverse events occurred in 7% receiving vosoritide versus 19% receiving placebo.

    Peptide
    Vosoritide
    Outcome
    DecreaseSerious adverse events
    Study

    n: not reported in source

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Adverse events · Whole body
    Studied dose
    30.0 μg/kg for ages 0-23 months; 15.0 μg/kg for ages 24-59 months · subcutaneous · 52 weeksAs given in this study (human).

    Measurements

    Participants with serious adverse events
    7%
    Vosoritide group, three participants
    Participants with serious adverse events
    19%
    Placebo group, six participants

    Source sentences

    Summary
    Serious adverse events occurred in three (7%) participants in the vosoritide group (decreased oxygen saturation, respiratory syncytial virus bronchiolitis and sudden infant death syndrome, and pneumonia) and six (19%) participants in the placebo group
    1 context sentence
    Summary · context
    This double-blind, randomised, placebo-controlled, phase 2 trial was done in 16 hospitals across Australia, Japan, the UK, and the USA.
  6. 06HumanNon-randomized trialSafety outcome

    In young children with achondroplasia receiving vosoritide, decreased oxygen saturation occurred as a serious adverse event.

    In plain wordsDecreased oxygen saturation occurred as a serious adverse event in young children receiving vosoritide.

    Peptide
    Vosoritide
    Outcome
    Not applicableDecreased oxygen saturation
    Study

    n: not reported in source

    Analysis
    Primary
    Outcome group
    Adverse events · Respiratory
    Studied dose
    30.0 μg/kg for ages 0-23 months; 15.0 μg/kg for ages 24-59 months · subcutaneous · 52 weeksAs given in this study (human).

    Source sentences

    Summary
    Serious adverse events occurred in three (7%) participants in the vosoritide group (decreased oxygen saturation, respiratory syncytial virus bronchiolitis and sudden infant death syndrome, and pneumonia)
    1 context sentence
    Summary · context
    This double-blind, randomised, placebo-controlled, phase 2 trial was done in 16 hospitals across Australia, Japan, the UK, and the USA.
  7. 07HumanNon-randomized trialSafety outcome

    In young children with achondroplasia receiving vosoritide, respiratory syncytial virus bronchiolitis occurred as a serious adverse event.

    In plain wordsRespiratory syncytial virus bronchiolitis occurred as a serious adverse event in young children receiving vosoritide.

    Peptide
    Vosoritide
    Outcome
    Not applicableRespiratory syncytial virus bronchiolitis
    Study

    n: not reported in source

    Analysis
    Primary
    Outcome group
    Adverse events · Respiratory
    Studied dose
    30.0 μg/kg for ages 0-23 months; 15.0 μg/kg for ages 24-59 months · subcutaneous · 52 weeksAs given in this study (human).

    Source sentences

    Summary
    Serious adverse events occurred in three (7%) participants in the vosoritide group (decreased oxygen saturation, respiratory syncytial virus bronchiolitis and sudden infant death syndrome, and pneumonia)
    1 context sentence
    Summary · context
    This double-blind, randomised, placebo-controlled, phase 2 trial was done in 16 hospitals across Australia, Japan, the UK, and the USA.
  8. 08HumanNon-randomized trialSafety outcome

    In young children with achondroplasia receiving vosoritide, sudden infant death syndrome occurred as a serious adverse event.

    In plain wordsSudden infant death syndrome occurred in the vosoritide group.

    Peptide
    Vosoritide
    Outcome
    Not applicableSudden infant death syndrome
    Study

    n: not reported in source

    Analysis
    Primary
    Outcome group
    Mortality · Whole body
    Studied dose
    30.0 μg/kg for ages 0-23 months; 15.0 μg/kg for ages 24-59 months · subcutaneous · 52 weeksAs given in this study (human).

    Source sentences

    Summary
    Serious adverse events occurred in three (7%) participants in the vosoritide group (decreased oxygen saturation, respiratory syncytial virus bronchiolitis and sudden infant death syndrome, and pneumonia)
    1 context sentence
    Summary · context
    This double-blind, randomised, placebo-controlled, phase 2 trial was done in 16 hospitals across Australia, Japan, the UK, and the USA.
  9. 09HumanNon-randomized trialSafety outcome

    In young children with achondroplasia receiving vosoritide, pneumonia occurred as a serious adverse event.

    In plain wordsPneumonia occurred as a serious adverse event in young children receiving vosoritide.

    Peptide
    Vosoritide
    Outcome
    Not applicablePneumonia
    Study

    n: not reported in source

    Analysis
    Primary
    Outcome group
    Adverse events · Respiratory
    Studied dose
    30.0 μg/kg for ages 0-23 months; 15.0 μg/kg for ages 24-59 months · subcutaneous · 52 weeksAs given in this study (human).

    Source sentences

    Summary
    Serious adverse events occurred in three (7%) participants in the vosoritide group (decreased oxygen saturation, respiratory syncytial virus bronchiolitis and sudden infant death syndrome, and pneumonia)
    1 context sentence
    Summary · context
    This double-blind, randomised, placebo-controlled, phase 2 trial was done in 16 hospitals across Australia, Japan, the UK, and the USA.

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 · 1 with posted results · source ClinicalTrials.gov (opens in a new tab)

CompletedPhase 2Results postedNCT03583697

A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

BioMarin Pharmaceuticaln = 7513 Jun 2018 to 26 Jan 2022

Achondroplasia

Trial pageClinicalTrials.govUpdated 13 Jun 2024

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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