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Study · Non-randomized trial · n = 35 · 2019

8 results

C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia.

Savarirayan R, Irving M, Bacino CA, Bostwick B, Charrow J, Cormier-Daire V, Le Quan Sang KH, Dickson P, Harmatz P, Phillips J, Owen N, Cherukuri A and 7 more

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Jayaram K, Jeha GS, Larimore K, Chan ML, Huntsman Labed A, Day J, Hoover-Fong J

The New England journal of medicine18 Jun 2019Cited by 162 (Europe PMC)

PMID31269546 (opens in a new tab)DOI10.1056/nejmoa1813446 (opens in a new tab)PepBankPUB-31269546
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 study8

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

  1. 01HumanNon-randomized trialSafety outcome

    In children with achondroplasia, adverse events occurred in all vosoritide recipients during treatment in the dose-finding and extension studies.

    In plain wordsIn children with achondroplasia, all vosoritide recipients had adverse events during treatment.

    Peptide
    Vosoritide
    Outcome
    Not applicableAny adverse event
    Study

    n = 35

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Measurements

    Patients with adverse events
    35 patients
    vosoritide
    Adverse event proportion
    100%
    vosoritide

    Source sentences

    Summary
    During the treatment periods in the dose-finding and extension studies, adverse events occurred in 35 of 35 patients (100%), and serious adverse events occurred in 4 of 35 patients (11%).
    1 context sentence
    Summary · context
    In a multinational, phase 2, dose-finding study and extension study, we evaluated the safety and side-effect profile of vosoritide in children (5 to 14 years of age) with achondroplasia.
  2. 02HumanNon-randomized trialSafety outcome

    In children with achondroplasia, serious adverse events occurred in 4 of 35 vosoritide recipients during the treatment periods.

    In plain wordsIn children with achondroplasia, 4 of 35 vosoritide recipients had serious adverse events during treatment.

    Peptide
    Vosoritide
    Outcome
    Not applicableSerious adverse events
    Study

    n = 35

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Measurements

    Patients with serious adverse events
    4 patients
    vosoritide
    Serious adverse event proportion
    11%
    vosoritide

    Source sentences

    Summary
    During the treatment periods in the dose-finding and extension studies, adverse events occurred in 35 of 35 patients (100%), and serious adverse events occurred in 4 of 35 patients (11%).
    1 context sentence
    Summary · context
    In a multinational, phase 2, dose-finding study and extension study, we evaluated the safety and side-effect profile of vosoritide in children (5 to 14 years of age) with achondroplasia.
  3. 03HumanNon-randomized trialSafety outcome

    In children with achondroplasia, 6 patients discontinued vosoritide therapy.

    In plain wordsIn children with achondroplasia, 6 patients stopped vosoritide therapy.

    Peptide
    Vosoritide
    Outcome
    Not applicableTreatment discontinuation
    Study

    n = 35

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Measurements

    Treatment discontinuations
    6 patients
    vosoritide

    Source sentences

    Summary
    Therapy was discontinued in 6 patients (in 1 because of an adverse event).
    1 context sentence
    Summary · context
    In a multinational, phase 2, dose-finding study and extension study, we evaluated the safety and side-effect profile of vosoritide in children (5 to 14 years of age) with achondroplasia.
  4. 04HumanNon-randomized trialSafety outcome

    In children with achondroplasia, 1 patient discontinued vosoritide therapy because of an adverse event.

    In plain wordsIn children with achondroplasia, 1 patient stopped vosoritide because of an adverse event.

    Peptide
    Vosoritide
    Outcome
    Not applicableDiscontinuation because of an adverse event
    Study

    n = 35

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Measurements

    Discontinuation because of an adverse event
    1 patient
    vosoritide

    Source sentences

    Summary
    Therapy was discontinued in 6 patients (in 1 because of an adverse event).
    1 context sentence
    Summary · context
    In a multinational, phase 2, dose-finding study and extension study, we evaluated the safety and side-effect profile of vosoritide in children (5 to 14 years of age) with achondroplasia.
  5. 05HumanNon-randomized trialSafety outcome

    In children with achondroplasia, once-daily subcutaneous vosoritide was associated with a side-effect profile that appeared generally mild.

    In plain wordsIn children with achondroplasia, side effects during once-daily subcutaneous vosoritide treatment appeared generally mild.

    Peptide
    Vosoritide
    Outcome
    Not applicableGenerally mild side-effect profile
    Study

    n = 35

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    In children with achondroplasia, once-daily subcutaneous administration of vosoritide was associated with a side-effect profile that appeared generally mild.
    1 context sentence
    Summary · context
    In a multinational, phase 2, dose-finding study and extension study, we evaluated the safety and side-effect profile of vosoritide in children (5 to 14 years of age) with achondroplasia.
  6. 06HumanNon-randomized trialEfficacy outcome

    In children with achondroplasia, vosoritide showed a dose-dependent increase in annualized growth velocity up to 15.0 μg per kilogram during the first 6 months.

    In plain wordsIn children with achondroplasia, growth velocity increased with vosoritide dose up to 15.0 μg per kilogram during the first 6 months.

    Peptide
    Vosoritide
    Outcome
    IncreaseDose-dependent annualized growth velocity increase
    Study

    n: not reported in source

    Outcome group
    Linear growth · Musculoskeletal
    Studied dose
    2.5 to 15.0 μg per kilogram once daily · subcutaneous · 6 monthsAs given in this study (human).

    Source sentences

    Summary
    During the first 6 months of treatment, a dose-dependent increase in the annualized growth velocity was observed with vosoritide up to a dose of 15.0 μg per kilogram
    2 context sentences
    Summary · context
    A total of 35 children were enrolled in four sequential cohorts to receive vosoritide at a once-daily subcutaneous dose of 2.5 μg per kilogram of body weight (8 patients in cohort 1), 7.5 μg per kilogram (8 patients in cohort 2), 15.0 μg per kilogram (10 patients in cohort 3), or 30.0 μg per kilogra…
    Summary · context
    In a multinational, phase 2, dose-finding study and extension study, we evaluated the safety and side-effect profile of vosoritide in children (5 to 14 years of age) with achondroplasia.
  7. 07HumanNon-randomized trialEfficacy outcome

    In children with achondroplasia, vosoritide at 15.0 μg per kilogram showed a sustained increase in annualized growth velocity for up to 42 months.

    In plain wordsIn children with achondroplasia, vosoritide at 15.0 μg per kilogram showed sustained growth velocity increases for up to 42 months.

    Peptide
    Vosoritide
    Outcome
    IncreaseSustained annualized growth velocity increase at 15.0 μg per kilogram
    Study

    n: not reported in source

    Outcome group
    Linear growth · Musculoskeletal
    Studied dose
    15.0 μg per kilogram once daily · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    and a sustained increase in the annualized growth velocity was observed at doses of 15.0 and 30.0 μg per kilogram for up to 42 months.
    2 context sentences
    Summary · context
    A total of 35 children were enrolled in four sequential cohorts to receive vosoritide at a once-daily subcutaneous dose of 2.5 μg per kilogram of body weight (8 patients in cohort 1), 7.5 μg per kilogram (8 patients in cohort 2), 15.0 μg per kilogram (10 patients in cohort 3), or 30.0 μg per kilogra…
    Summary · context
    In a multinational, phase 2, dose-finding study and extension study, we evaluated the safety and side-effect profile of vosoritide in children (5 to 14 years of age) with achondroplasia.
  8. 08HumanNon-randomized trialEfficacy outcome

    In children with achondroplasia, vosoritide at 30.0 μg per kilogram showed a sustained increase in annualized growth velocity for up to 42 months.

    In plain wordsIn children with achondroplasia, vosoritide at 30.0 μg per kilogram showed sustained growth velocity increases for up to 42 months.

    Peptide
    Vosoritide
    Outcome
    IncreaseSustained annualized growth velocity increase at 30.0 μg per kilogram
    Study

    n: not reported in source

    Outcome group
    Linear growth · Musculoskeletal
    Studied dose
    30.0 μg per kilogram once daily · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    and a sustained increase in the annualized growth velocity was observed at doses of 15.0 and 30.0 μg per kilogram for up to 42 months.
    2 context sentences
    Summary · context
    A total of 35 children were enrolled in four sequential cohorts to receive vosoritide at a once-daily subcutaneous dose of 2.5 μg per kilogram of body weight (8 patients in cohort 1), 7.5 μg per kilogram (8 patients in cohort 2), 15.0 μg per kilogram (10 patients in cohort 3), or 30.0 μg per kilogra…
    Summary · context
    In a multinational, phase 2, dose-finding study and extension study, we evaluated the safety and side-effect profile of vosoritide in children (5 to 14 years of age) with achondroplasia.

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 2NCT02724228

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

BioMarin Pharmaceuticaln = 3026 Jan 2016 to est. Feb 2028

Achondroplasia

Trial pageClinicalTrials.govUpdated 13 Mar 2026
CompletedPhase 2Results postedNCT02055157

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

BioMarin Pharmaceuticaln = 3513 Jan 2014 to 2 Oct 2017

Achondroplasia

Trial pageClinicalTrials.govUpdated 15 Jan 2021

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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