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Study · Non-randomized trial · n = 38 · 2024

8 results

Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life.

Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Mohnike K, Prada CE, Kubota T, Arundel P, Leiva-Gea A and 5 more

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Rowell R, Low A, Sabir I, Huntsman-Labed A, Day J

Genetics in medicine : official journal of the American College of Medical Genetics18 Sept 2024Cited by 15 (Europe PMC)

PMID39305160 (opens in a new tab)DOI10.1016/j.gim.2024.101274 (opens in a new tab)PepBankPUB-39305160
Publisher pagePubMedAsk about this study
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Summary

PurposeEvaluate the impact of vosoritide on health-related quality of life in children with achondroplasia.MethodsParticipants received vosoritide (15 μg/kg/day) in an extension trial (NCT03424018) after having participated in a placebo-controlled trial (NCT03197766).ResultsThe population comprised 119 participants (mean [SD] age 9.7 [2.6] years). Mean treatment duration was 4 (0.78) years. At year 3, the largest mean (SD) changes were observed in the Quality of Life of Short Stature Youth physical score (5.99 [19.41], caregiver reported; 6.32 [20.15], self-reported) and social score (2.85 [8.29] and 6.76 [22.64], respectively). Changes were greatest in participants with ≥1 SD increase in height z-score (physical: 11.36 [19.51], caregiver-reported [n = 38]; 8.48 [21.83], self-reported [n = 28]) (social: 5.84 [15.45] and 9.79 [22.80], respectively). To determine how domain scores may change with age in untreated persons, models were produced using observational/untreated-person data. A 1-year increase in age was associated with a change of 0.16 (SE, 0.55) and 0.16 (0.50), for caregiver-reported physical and social domain scores, respectively. Self-reported scores changed by 1.45 (0.71) and 1.92 (0.77), respectively.ConclusionThese data suggest that after 3 years of treatment, vosoritide demonstrates a positive effect on physical and social functioning among children with achondroplasia, particularly in children with a more pronounced change in height z-score.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study8

8 results from studies that gave the peptide. 8 results from people.

  1. 01HumanNon-randomized trialEfficacy outcome

    In children with achondroplasia receiving vosoritide, caregiver-reported physical score changed by 5.99 (SD 19.41) at year 3 from baseline.

    In plain wordsIn children receiving vosoritide, caregiver-reported physical scores increased from baseline at year 3.

    Peptide
    Vosoritide
    Outcome
    IncreaseCaregiver-reported physical score at year 3
    Study

    n: not reported in source

    Comparator
    Baseline
    Outcome group
    Quality of life · Whole body
    Studied dose
    15 μg/kg/dayAs given in this study (human).

    Measurements

    Mean change in caregiver-reported QoLISSY physical score
    5.99 points
    Year 3, caregiver-reported
    SD of change in caregiver-reported QoLISSY physical score
    19.41 points
    Year 3, caregiver-reported

    Source sentences

    Summary
    At year 3, the largest mean (SD) changes were observed in the Quality of Life of Short Stature Youth physical score (5.99 [19.41], caregiver reported; 6.32 [20.15], self-reported) and social score (2.85 [8.29] and 6.76 [22.64], respectively).
    2 context sentences
    Summary · context
    Participants received vosoritide (15 μg/kg/day) in an extension trial (NCT03424018) after having participated in a placebo-controlled trial (NCT03197766).
    Summary · context
    The population comprised 119 participants (mean [SD] age 9.7 [2.6] years).
  2. 02HumanNon-randomized trialEfficacy outcome

    In children with achondroplasia receiving vosoritide, self-reported physical score changed by 6.32 (SD 20.15) at year 3 from baseline.

    In plain wordsIn children receiving vosoritide, self-reported physical scores increased from baseline at year 3.

    Peptide
    Vosoritide
    Outcome
    IncreaseSelf-reported physical score at year 3
    Study

    n: not reported in source

    Comparator
    Baseline
    Outcome group
    Quality of life · Whole body
    Studied dose
    15 μg/kg/dayAs given in this study (human).

    Measurements

    Mean change in self-reported QoLISSY physical score
    6.32 points
    Year 3, self-reported
    SD of change in self-reported QoLISSY physical score
    20.15 points
    Year 3, self-reported

    Source sentences

    Summary
    At year 3, the largest mean (SD) changes were observed in the Quality of Life of Short Stature Youth physical score (5.99 [19.41], caregiver reported; 6.32 [20.15], self-reported) and social score (2.85 [8.29] and 6.76 [22.64], respectively).
    2 context sentences
    Summary · context
    Participants received vosoritide (15 μg/kg/day) in an extension trial (NCT03424018) after having participated in a placebo-controlled trial (NCT03197766).
    Summary · context
    The population comprised 119 participants (mean [SD] age 9.7 [2.6] years).
  3. 03HumanNon-randomized trialEfficacy outcome

    In children with achondroplasia receiving vosoritide, caregiver-reported social score changed by 2.85 (SD 8.29) at year 3 from baseline.

    In plain wordsIn children receiving vosoritide, caregiver-reported social scores increased from baseline at year 3.

    Peptide
    Vosoritide
    Outcome
    IncreaseCaregiver-reported social score at year 3
    Study

    n: not reported in source

    Comparator
    Baseline
    Outcome group
    Quality of life · Whole body
    Studied dose
    15 μg/kg/dayAs given in this study (human).

    Measurements

    Mean change in caregiver-reported QoLISSY social score
    2.85 points
    Year 3, caregiver-reported
    SD of change in caregiver-reported QoLISSY social score
    8.29 points
    Year 3, caregiver-reported

    Source sentences

    Summary
    At year 3, the largest mean (SD) changes were observed in the Quality of Life of Short Stature Youth physical score (5.99 [19.41], caregiver reported; 6.32 [20.15], self-reported) and social score (2.85 [8.29] and 6.76 [22.64], respectively).
    2 context sentences
    Summary · context
    Participants received vosoritide (15 μg/kg/day) in an extension trial (NCT03424018) after having participated in a placebo-controlled trial (NCT03197766).
    Summary · context
    The population comprised 119 participants (mean [SD] age 9.7 [2.6] years).
  4. 04HumanNon-randomized trialEfficacy outcome

    In children with achondroplasia receiving vosoritide, self-reported social score changed by 6.76 (SD 22.64) at year 3 from baseline.

    In plain wordsIn children receiving vosoritide, self-reported social scores increased from baseline at year 3.

    Peptide
    Vosoritide
    Outcome
    IncreaseSelf-reported social score at year 3
    Study

    n: not reported in source

    Comparator
    Baseline
    Outcome group
    Quality of life · Whole body
    Studied dose
    15 μg/kg/dayAs given in this study (human).

    Measurements

    Mean change in self-reported QoLISSY social score
    6.76 points
    Year 3, self-reported
    SD of change in self-reported QoLISSY social score
    22.64 points
    Year 3, self-reported

    Source sentences

    Summary
    At year 3, the largest mean (SD) changes were observed in the Quality of Life of Short Stature Youth physical score (5.99 [19.41], caregiver reported; 6.32 [20.15], self-reported) and social score (2.85 [8.29] and 6.76 [22.64], respectively).
    2 context sentences
    Summary · context
    Participants received vosoritide (15 μg/kg/day) in an extension trial (NCT03424018) after having participated in a placebo-controlled trial (NCT03197766).
    Summary · context
    The population comprised 119 participants (mean [SD] age 9.7 [2.6] years).
  5. 05HumanObservational studyEfficacy outcome

    In vosoritide-treated children with ≥1 SD height z-score increase, caregiver-reported physical score changed by 11.36 (SD 19.51).

    In plain wordsIn children receiving vosoritide with larger height responses, caregiver-reported physical scores increased from baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseCaregiver-reported physical score in height-response subgroup
    Study

    n = 38

    Comparator
    Baseline
    Outcome group
    Quality of life · Whole body
    Studied dose
    15 μg/kg/dayAs given in this study (human).

    Measurements

    Mean change in caregiver-reported physical score
    11.36 points
    Participants with >=1 SD height z-score increase, caregiver-reported, n=38
    SD of change in caregiver-reported physical score
    19.51 points
    Participants with >=1 SD height z-score increase, caregiver-reported, n=38

    Source sentences

    Summary
    Changes were greatest in participants with ≥1 SD increase in height z-score (physical: 11.36 [19.51], caregiver-reported [n = 38]
    2 context sentences
    Summary · context
    Participants received vosoritide (15 μg/kg/day) in an extension trial (NCT03424018) after having participated in a placebo-controlled trial (NCT03197766).
    Summary · context
    The population comprised 119 participants (mean [SD] age 9.7 [2.6] years).
  6. 06HumanObservational studyEfficacy outcome

    In vosoritide-treated children with ≥1 SD height z-score increase, self-reported physical score changed by 8.48 (SD 21.83).

    In plain wordsIn children receiving vosoritide with larger height responses, self-reported physical scores increased from baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseSelf-reported physical score in height-response subgroup
    Study

    n = 28

    Comparator
    Baseline
    Outcome group
    Quality of life · Whole body
    Studied dose
    15 μg/kg/dayAs given in this study (human).

    Measurements

    Mean change in self-reported physical score
    8.48 points
    Participants with >=1 SD height z-score increase, self-reported, n=28
    SD of change in self-reported physical score
    21.83 points
    Participants with >=1 SD height z-score increase, self-reported, n=28

    Source sentences

    Summary
    Changes were greatest in participants with ≥1 SD increase in height z-score (physical: 11.36 [19.51], caregiver-reported [n = 38]; 8.48 [21.83], self-reported [n = 28])
    2 context sentences
    Summary · context
    Participants received vosoritide (15 μg/kg/day) in an extension trial (NCT03424018) after having participated in a placebo-controlled trial (NCT03197766).
    Summary · context
    The population comprised 119 participants (mean [SD] age 9.7 [2.6] years).
  7. 07HumanObservational studyEfficacy outcome

    In vosoritide-treated children with ≥1 SD height z-score increase, caregiver-reported social score changed by 5.84 (SD 15.45).

    In plain wordsIn children receiving vosoritide with larger height responses, caregiver-reported social scores increased from baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseCaregiver-reported social score in height-response subgroup
    Study

    n: not reported in source

    Comparator
    Baseline
    Outcome group
    Quality of life · Whole body
    Studied dose
    15 μg/kg/dayAs given in this study (human).

    Measurements

    Mean change in caregiver-reported social score
    5.84 points
    Participants with >=1 SD height z-score increase, caregiver-reported
    SD of change in caregiver-reported social score
    15.45 points
    Participants with >=1 SD height z-score increase, caregiver-reported

    Source sentences

    Summary
    Changes were greatest in participants with ≥1 SD increase in height z-score (physical: 11.36 [19.51], caregiver-reported [n = 38]; 8.48 [21.83], self-reported [n = 28]) (social: 5.84 [15.45] and 9.79 [22.80], respectively).
    2 context sentences
    Summary · context
    Participants received vosoritide (15 μg/kg/day) in an extension trial (NCT03424018) after having participated in a placebo-controlled trial (NCT03197766).
    Summary · context
    The population comprised 119 participants (mean [SD] age 9.7 [2.6] years).
  8. 08HumanObservational studyEfficacy outcome

    In vosoritide-treated children with ≥1 SD height z-score increase, self-reported social score changed by 9.79 (SD 22.80).

    In plain wordsIn children receiving vosoritide with larger height responses, self-reported social scores increased from baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseSelf-reported social score in height-response subgroup
    Study

    n: not reported in source

    Comparator
    Baseline
    Outcome group
    Quality of life · Whole body
    Studied dose
    15 μg/kg/dayAs given in this study (human).

    Measurements

    Mean change in self-reported social score
    9.79 points
    Participants with >=1 SD height z-score increase, self-reported
    SD of change in self-reported social score
    22.8 points
    Participants with >=1 SD height z-score increase, self-reported

    Source sentences

    Summary
    Changes were greatest in participants with ≥1 SD increase in height z-score (physical: 11.36 [19.51], caregiver-reported [n = 38]; 8.48 [21.83], self-reported [n = 28]) (social: 5.84 [15.45] and 9.79 [22.80], respectively).
    2 context sentences
    Summary · context
    Participants received vosoritide (15 μg/kg/day) in an extension trial (NCT03424018) after having participated in a placebo-controlled trial (NCT03197766).
    Summary · context
    The population comprised 119 participants (mean [SD] age 9.7 [2.6] years).

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

Registered trials2

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

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

Active, not recruitingPhase 3NCT03424018

An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

BioMarin Pharmaceuticaln = 11912 Dec 2017 to est. Jun 2031

Achondroplasia

Trial pageClinicalTrials.govUpdated 13 Mar 2026
CompletedPhase 3Results postedNCT03197766

A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

BioMarin Pharmaceuticaln = 12112 Dec 2016 to 30 Oct 2019

Achondroplasia

Trial pageClinicalTrials.govUpdated 2 Mar 2022

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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