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Study · Non-randomized trial · n = 24 · 2024

7 results

Vosoritide treatment for children with hypochondroplasia: a phase 2 trial.

Dauber A, Zhang A, Kanakatti Shankar R, Boucher K, McCarthy T, Shafaei N, Seaforth R, Castro MG, Dham N, Merchant N

EClinicalMedicine11 Apr 2024Cited by 19 (Europe PMC)

PMID38813446 (opens in a new tab)PMCIDPMC11133798 (opens in a new tab)DOI10.1016/j.eclinm.2024.102591 (opens in a new tab)PepBankPUB-38813446
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Summary

BackgroundHypochondroplasia is a rare autosomal dominant skeletal dysplasia due to activating variants in FGFR3. It presents with disproportionate short stature with a wide range of clinical severity. There are currently no approved medications to treat short stature in children with hypochondroplasia. Vosoritide is a C-type natriuretic peptide analog that was recently approved for improving growth in children with achondroplasia. We aimed to evaluate the safety and efficacy of vosoritide in children with hypochondroplasia.MethodsWe conducted a single-arm, phase 2, open-label trial at a single centre in the USA and enrolled 26 children with hypochondroplasia. The trial consists of a 6-month observation period to establish a baseline annualized growth velocity followed by a 12-month intervention period during which vosoritide is administered daily via subcutaneous injection at a dose of 15 μg/kg/day. The trial's co-primary endpoints included the incidence of adverse events and the change from baseline in age-sex standardized annualized growth velocity and height standardized deviation score (SDS) after 12 months of treatment. This trial is registered with ClinicalTrials.gov (NCT04219007).FindingsTwenty-four participants with a mean age of 5.86 years received vosoritide therapy. The first participant was enrolled on August 4, 2020, and the final participant completed the 18-month trial on September 8, 2023. Vosoritide was well tolerated with no treatment-related serious adverse events. Injection site reactions occurred in 83.3% of participants. No participants discontinued therapy due to an adverse event. Annualized growth velocity increased by 2.26 standard deviations (SD) and height SDS increased by 0.36 SD during the treatment period versus the observation period. Hypochondroplasia specific height SDS increased by 0.38 SD. There was a 1.81 cm/year increase in absolute annualized growth velocity.InterpretationVosoritide was safe and effective in increasing growth velocity in children with hypochondroplasia. Efficacy was similar to what has been reported in children with achondroplasia.FundingThis study was supported by an investigator-initiated grant from BioMarin Pharmaceutical.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study7

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

  1. 01HumanNon-randomized trialSafety outcome

    In children with hypochondroplasia receiving vosoritide, no treatment-related serious adverse events occurred.

    In plain wordsNo treatment-related serious adverse events occurred in children with hypochondroplasia receiving vosoritide.

    Peptide
    Vosoritide
    Outcome
    Not applicableTreatment-related serious adverse events
    Study

    n = 24

    Analysis
    Primary
    Outcome group
    Adverse events · Whole body
    Studied dose
    15 μg/kg/day · subcutaneous · 12 monthsAs given in this study (human).

    Source sentences

    Summary
    Vosoritide was well tolerated with no treatment-related serious adverse events.
    3 context sentences
    Summary · context
    We conducted a single-arm, phase 2, open-label trial at a single centre in the USA and enrolled 26 children with hypochondroplasia.
    Summary · context
    The trial consists of a 6-month observation period to establish a baseline annualized growth velocity followed by a 12-month intervention period during which vosoritide is administered daily via subcutaneous injection at a dose of 15 μg/kg/day.
    Summary · context
    Twenty-four participants with a mean age of 5.86 years received vosoritide therapy.
  2. 02HumanNon-randomized trialSafety outcome

    In children with hypochondroplasia receiving vosoritide, injection site reactions occurred in 83.3% of participants.

    In plain wordsInjection site reactions occurred in 83.3% of children with hypochondroplasia receiving vosoritide.

    Peptide
    Vosoritide
    Outcome
    Not applicableInjection site reactions
    Study

    n = 24

    Analysis
    Primary
    Outcome group
    Adverse events · Skin and wound
    Studied dose
    15 μg/kg/day · subcutaneous · 12 monthsAs given in this study (human).

    Measurements

    Participants with injection site reactions
    83.3%
    24 children on vosoritide

    Source sentences

    Summary
    Injection site reactions occurred in 83.3% of participants.
    3 context sentences
    Summary · context
    We conducted a single-arm, phase 2, open-label trial at a single centre in the USA and enrolled 26 children with hypochondroplasia.
    Summary · context
    The trial consists of a 6-month observation period to establish a baseline annualized growth velocity followed by a 12-month intervention period during which vosoritide is administered daily via subcutaneous injection at a dose of 15 μg/kg/day.
    Summary · context
    Twenty-four participants with a mean age of 5.86 years received vosoritide therapy.
  3. 03HumanNon-randomized trialSafety outcome

    In children with hypochondroplasia receiving vosoritide, no participants discontinued therapy because of an adverse event.

    In plain wordsNo children with hypochondroplasia stopped vosoritide because of an adverse event.

    Peptide
    Vosoritide
    Outcome
    Not applicableAdverse event discontinuations
    Study

    n = 24

    Analysis
    Primary
    Outcome group
    Adverse events · Whole body
    Studied dose
    15 μg/kg/day · subcutaneous · 12 monthsAs given in this study (human).

    Source sentences

    Summary
    No participants discontinued therapy due to an adverse event.
    3 context sentences
    Summary · context
    We conducted a single-arm, phase 2, open-label trial at a single centre in the USA and enrolled 26 children with hypochondroplasia.
    Summary · context
    The trial consists of a 6-month observation period to establish a baseline annualized growth velocity followed by a 12-month intervention period during which vosoritide is administered daily via subcutaneous injection at a dose of 15 μg/kg/day.
    Summary · context
    Twenty-four participants with a mean age of 5.86 years received vosoritide therapy.
  4. 04HumanNon-randomized trialEfficacy outcome

    In children with hypochondroplasia, standardized annualized growth velocity increased by 2.26 SD during vosoritide treatment versus observation.

    In plain wordsIn children with hypochondroplasia, standardized growth velocity increased during vosoritide treatment compared with observation.

    Peptide
    Vosoritide
    Outcome
    IncreaseStandardized annualized growth velocity
    Study

    n = 24

    Comparator
    Observation period
    Analysis
    Primary
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 μg/kg/day · subcutaneous · 12 monthsAs given in this study (human).

    Measurements

    Change in standardized annualized growth velocity
    2.26 SD
    Treatment vs observation period

    Source sentences

    Summary
    Annualized growth velocity increased by 2.26 standard deviations (SD) and height SDS increased by 0.36 SD during the treatment period versus the observation period.
    3 context sentences
    Summary · context
    We conducted a single-arm, phase 2, open-label trial at a single centre in the USA and enrolled 26 children with hypochondroplasia.
    Summary · context
    The trial consists of a 6-month observation period to establish a baseline annualized growth velocity followed by a 12-month intervention period during which vosoritide is administered daily via subcutaneous injection at a dose of 15 μg/kg/day.
    Summary · context
    Twenty-four participants with a mean age of 5.86 years received vosoritide therapy.
  5. 05HumanNon-randomized trialEfficacy outcome

    In children with hypochondroplasia, height SDS increased by 0.36 SD during vosoritide treatment versus observation.

    In plain wordsIn children with hypochondroplasia, height scores increased during vosoritide treatment compared with observation.

    Peptide
    Vosoritide
    Outcome
    IncreaseHeight SDS
    Study

    n = 24

    Comparator
    Observation period
    Analysis
    Primary
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 μg/kg/day · subcutaneous · 12 monthsAs given in this study (human).

    Measurements

    Change in height SDS
    0.36 SD
    Treatment vs observation period

    Source sentences

    Summary
    Annualized growth velocity increased by 2.26 standard deviations (SD) and height SDS increased by 0.36 SD during the treatment period versus the observation period.
    3 context sentences
    Summary · context
    We conducted a single-arm, phase 2, open-label trial at a single centre in the USA and enrolled 26 children with hypochondroplasia.
    Summary · context
    The trial consists of a 6-month observation period to establish a baseline annualized growth velocity followed by a 12-month intervention period during which vosoritide is administered daily via subcutaneous injection at a dose of 15 μg/kg/day.
    Summary · context
    Twenty-four participants with a mean age of 5.86 years received vosoritide therapy.
  6. 06HumanNon-randomized trialEfficacy outcome

    In children with hypochondroplasia receiving vosoritide, disease-specific height SDS increased by 0.38 SD.

    In plain wordsIn children with hypochondroplasia receiving vosoritide, disease-specific height scores increased from baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseHypochondroplasia-specific height SDS
    Study

    n = 24

    Comparator
    Observation period
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 μg/kg/day · subcutaneous · 12 monthsAs given in this study (human).

    Measurements

    Change in hypochondroplasia-specific height SDS
    0.38 SD
    Vosoritide treatment

    Source sentences

    Summary
    Hypochondroplasia specific height SDS increased by 0.38 SD.
    3 context sentences
    Summary · context
    We conducted a single-arm, phase 2, open-label trial at a single centre in the USA and enrolled 26 children with hypochondroplasia.
    Summary · context
    The trial consists of a 6-month observation period to establish a baseline annualized growth velocity followed by a 12-month intervention period during which vosoritide is administered daily via subcutaneous injection at a dose of 15 μg/kg/day.
    Summary · context
    Twenty-four participants with a mean age of 5.86 years received vosoritide therapy.
  7. 07HumanNon-randomized trialEfficacy outcome

    In children with hypochondroplasia receiving vosoritide, absolute annualized growth velocity increased by 1.81 cm/year.

    In plain wordsIn children with hypochondroplasia receiving vosoritide, absolute growth velocity increased compared with observation.

    Peptide
    Vosoritide
    Outcome
    IncreaseAbsolute annualized growth velocity
    Study

    n = 24

    Comparator
    Observation period
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 μg/kg/day · subcutaneous · 12 monthsAs given in this study (human).

    Measurements

    Change in absolute annualized growth velocity
    1.81 cm/year
    Vosoritide treatment

    Source sentences

    Summary
    There was a 1.81 cm/year increase in absolute annualized growth velocity.
    3 context sentences
    Summary · context
    We conducted a single-arm, phase 2, open-label trial at a single centre in the USA and enrolled 26 children with hypochondroplasia.
    Summary · context
    The trial consists of a 6-month observation period to establish a baseline annualized growth velocity followed by a 12-month intervention period during which vosoritide is administered daily via subcutaneous injection at a dose of 15 μg/kg/day.
    Summary · context
    Twenty-four participants with a mean age of 5.86 years received vosoritide therapy.

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 ongoing · source ClinicalTrials.gov (opens in a new tab)

Active, not recruitingPhase 2NCT04219007

Vosoritide for Selected Genetic Causes of Short Stature

Andrew Daubern = 564 Aug 2020 to est. 1 Jun 2035

Short Stature

Trial pageClinicalTrials.govUpdated 18 Mar 2026

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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