Study · Non-randomized trial · n = 119 · 2025

9 results

Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study.

Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Palm K, Prada CE, Kubota T, Arundel P, Kotani Y and 9 more

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Leiva-Gea A, Bober MB, Hecht JT, Legare JM, Lawrinson S, Low A, Sabir I, Huntsman-Labed A, Day JRS

Med (New York, N.Y.)30 Dec 2024Cited by 15 (Europe PMC)

PMID39740666DOI10.1016/j.medj.2024.11.019PepBankPUB-39740666

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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 study9

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

01HumanNon-randomized trialSafety outcome
Vosoritide had a favorable safety profile in children with achondroplasia with up to 6 years of continuous treatment and 464.05 person years of exposure.
In plain wordsVosoritide had a favorable safety profile in children with achondroplasia during continuous treatment for up to 6 years.
Peptide
Vosoritide
Outcome
Not applicableSafety profile
Study
n = 119
Outcome group
Adverse events · Whole body
Studied dose
15 μg/kg daily · up to 6 yearsAs given in this study (human).
Source sentences
Summary
Vosoritide had a favorable safety profile with continuous treatment for up to 6 years (464.05 person years of exposure).
2 context sentences(design, population, supporting numbers)
Summary · context
After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials.gov: NCT03197766), participants were eligible to continue treatment in an open-label extension (ClinicalTrials.gov: NCT03424018) wherein all received 15 μg/kg vosoritide daily.
Summary · context
Data from the CLARITY achondroplasia study provided an external untreated control population and reference data.
Report a problemFND-3974066607
02HumanNon-randomized trialSafety outcome
No long-term harms were observed in children receiving vosoritide.
In plain wordsNo long-term harms were observed in children receiving vosoritide.
Peptide
Vosoritide
Outcome
Not applicableLong-term harms
Study
n = 119
Outcome group
Adverse events · Whole body
Studied dose
15 μg/kg daily · up to 6 yearsAs given in this study (human).
Source sentences
Summary
No long-term harms or deaths were observed.
2 context sentences(design, population, supporting numbers)
Summary · context
After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials.gov: NCT03197766), participants were eligible to continue treatment in an open-label extension (ClinicalTrials.gov: NCT03424018) wherein all received 15 μg/kg vosoritide daily.
Summary · context
Data from the CLARITY achondroplasia study provided an external untreated control population and reference data.
Report a problemFND-3974066608
03HumanNon-randomized trialSafety outcome
No deaths were observed in children receiving vosoritide.
In plain wordsNo deaths were observed in children receiving vosoritide.
Peptide
Vosoritide
Outcome
Not applicableDeaths
Study
n = 119
Outcome group
Mortality · Whole body
Studied dose
15 μg/kg daily · up to 6 yearsAs given in this study (human).
Source sentences
Summary
No long-term harms or deaths were observed.
2 context sentences(design, population, supporting numbers)
Summary · context
After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials.gov: NCT03197766), participants were eligible to continue treatment in an open-label extension (ClinicalTrials.gov: NCT03424018) wherein all received 15 μg/kg vosoritide daily.
Summary · context
Data from the CLARITY achondroplasia study provided an external untreated control population and reference data.
Report a problemFND-3974066609
04HumanNon-randomized trialEfficacy outcome
Before puberty, children with achondroplasia receiving vosoritide had annualized growth velocity similar to the average-stature population.
In plain wordsBefore puberty, children with achondroplasia receiving vosoritide grew at a similar rate to the average-stature population.
Peptide
Vosoritide
Outcome
Direction not reportedPrepubertal annualized growth velocity
Study
n: not reported in source
Comparator
Average-stature population
Outcome group
Linear growth · Musculoskeletal
Studied dose
15 μg/kg dailyAs given in this study (human).
Source sentences
Summary
Annualized growth velocity with vosoritide was similar to the average-stature population before puberty.
2 context sentences(design, population, supporting numbers)
Summary · context
After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials.gov: NCT03197766), participants were eligible to continue treatment in an open-label extension (ClinicalTrials.gov: NCT03424018) wherein all received 15 μg/kg vosoritide daily.
Summary · context
Data from the CLARITY achondroplasia study provided an external untreated control population and reference data.
Report a problemFND-3974066601
05HumanNon-randomized trialEfficacy outcome
Across integer ages 6-16 years, vosoritide-treated boys had mean growth velocity differences of 1.84 (SD 0.38) cm/year versus untreated children.
In plain wordsVosoritide-treated boys had higher growth velocity than untreated children across ages 6-16 years.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity in boys
Study
n: not reported in source
Comparator
Untreated children
Outcome group
Linear growth · Musculoskeletal
Studied dose
15 μg/kg dailyAs given in this study (human).
Measurements
Mean difference
1.84 cm/year
boys
SD
0.38 cm/year
boys
Source sentences
Summary
The mean (SD) differences in annualized growth velocity across each integer age (6-16 years) between treated and untreated children were 1.84 (0.38) cm/year in boys and 1.44 (0.63) cm/year in girls.
2 context sentences(design, population, supporting numbers)
Summary · context
After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials.gov: NCT03197766), participants were eligible to continue treatment in an open-label extension (ClinicalTrials.gov: NCT03424018) wherein all received 15 μg/kg vosoritide daily.
Summary · context
Data from the CLARITY achondroplasia study provided an external untreated control population and reference data.
Report a problemFND-3974066602
06HumanNon-randomized trialEfficacy outcome
Across integer ages 6-16 years, vosoritide-treated girls had mean growth velocity differences of 1.44 (SD 0.63) cm/year versus untreated children.
In plain wordsVosoritide-treated girls had higher growth velocity than untreated children across ages 6-16 years.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity in girls
Study
n: not reported in source
Comparator
Untreated children
Outcome group
Linear growth · Musculoskeletal
Studied dose
15 μg/kg dailyAs given in this study (human).
Measurements
Mean difference
1.44 cm/year
girls
SD
0.63 cm/year
girls
Source sentences
Summary
The mean (SD) differences in annualized growth velocity across each integer age (6-16 years) between treated and untreated children were 1.84 (0.38) cm/year in boys and 1.44 (0.63) cm/year in girls.
2 context sentences(design, population, supporting numbers)
Summary · context
After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials.gov: NCT03197766), participants were eligible to continue treatment in an open-label extension (ClinicalTrials.gov: NCT03424018) wherein all received 15 μg/kg vosoritide daily.
Summary · context
Data from the CLARITY achondroplasia study provided an external untreated control population and reference data.
Report a problemFND-3974066603
07HumanNon-randomized trialEfficacy outcome
At three years, vosoritide-treated children gained 5.75 cm additional height versus untreated children (95% CI 4.93, 6.57).
In plain wordsAt three years, vosoritide-treated children gained 5.75 cm additional height compared with untreated children.
Peptide
Vosoritide
Outcome
IncreaseAdditional height gain at three years
Study
n: not reported in source
Comparator
Untreated children
Outcome group
Linear growth · Musculoskeletal
Studied dose
15 μg/kg dailyAs given in this study (human).
Measurements
Additional height gain
5.75 cm
95% CI
4.93 to 6.57 cm
Source sentences
Summary
Three-year comparisons of treated versus untreated children demonstrated an additional height gain of 5.75 cm (95% confidence interval [CI]: 4.93, 6.57) with vosoritide.
2 context sentences(design, population, supporting numbers)
Summary · context
After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials.gov: NCT03197766), participants were eligible to continue treatment in an open-label extension (ClinicalTrials.gov: NCT03424018) wherein all received 15 μg/kg vosoritide daily.
Summary · context
Data from the CLARITY achondroplasia study provided an external untreated control population and reference data.
Report a problemFND-3974066604
08HumanNon-randomized trialEfficacy outcome
Vosoritide-treated children assessed below age 11 (females) or 12 (males) showed significant improvement in body segment ratio versus untreated controls.
In plain wordsYounger vosoritide-treated children showed significant improvement in body segment ratio compared with age-matched untreated controls.
Peptide
Vosoritide
Outcome
Direction not reportedUpper-to-lower body segment ratio
Study
n: not reported in source
Comparator
Population-level age-matched untreated controls
Outcome group
Linear growth · Musculoskeletal
Studied dose
15 μg/kg daily · 3 yearsAs given in this study (human).
Measurements
p
0.0087
Source sentences
Summary
A significant improvement in upper-to-lower body segment ratio at 3 years of treatment was observed for participants with assessments at age <11 (females) and <12 years (males) versus population-level, age-matched, untreated controls (p = 0.0087).
2 context sentences(design, population, supporting numbers)
Summary · context
After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials.gov: NCT03197766), participants were eligible to continue treatment in an open-label extension (ClinicalTrials.gov: NCT03424018) wherein all received 15 μg/kg vosoritide daily.
Summary · context
Data from the CLARITY achondroplasia study provided an external untreated control population and reference data.
Report a problemFND-3974066605
09HumanNon-randomized trialEfficacy outcome
In children receiving vosoritide, arm span-to-standing height ratio remained consistent with untreated participants.
In plain wordsChildren receiving vosoritide had an arm span-to-height ratio consistent with untreated participants.
Peptide
Vosoritide
Outcome
Direction not reportedArm span-to-standing height ratio
Study
n: not reported in source
Comparator
Untreated participants
Outcome group
Linear growth · Musculoskeletal
Studied dose
15 μg/kg dailyAs given in this study (human).
Source sentences
Summary
The arm span-to-standing height ratio remained consistent with untreated participants.
2 context sentences(design, population, supporting numbers)
Summary · context
After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials.gov: NCT03197766), participants were eligible to continue treatment in an open-label extension (ClinicalTrials.gov: NCT03424018) wherein all received 15 μg/kg vosoritide daily.
Summary · context
Data from the CLARITY achondroplasia study provided an external untreated control population and reference data.
Report a problemFND-3974066606

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 page ClinicalTrials.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 page ClinicalTrials.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.

Version 2Current
28 Oct 2026, 02:03 UTC
Study results updated
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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