OR18-5 A Prospective Clinical Trial of Vosoritide in Selected Genetic Causes of Short Stature

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Journal of the Endocrine Society1 Nov 2022

PMCIDPMC9625806PepBankPUB-92378987

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Summary

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Text rights: CC BY-NC-ND. Results still quote short attributed sentences.

Results from this study11

11 results from studies that gave the peptide. 11 results from people.

01HumanNon-randomized trialEfficacy outcome
In children with genetic short stature, vosoritide increased median annualized growth velocity by 3.9 cm/yr (IQR 2.2, 4.6).
In plain wordsIn children with genetic short stature, vosoritide increased median annualized growth velocity by 3.9 cm/yr from baseline.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity
Study
n: not reported in source
Comparator
Baseline
Analysis
Primary
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15 mcg/kg/day · subcutaneousAs given in this study (human).
Measurements
Median increase in annualized growth velocity
3.9 cm/yr
From baseline
IQR of increase in annualized growth velocity
2.2 to 4.6 cm/yr
From baseline
Source sentences
Summary
Median increase in AGV was 3.9 cm/yr (IQR 2.2, 4.6).
2 context sentences(design, population, supporting numbers)
Summary · context
14 subjects have initiated on vosoritide, and 10 completed 6 months of therapy.
Summary · context
Subjects are followed for a 6-month observation period to establish a baseline annualized growth velocity (AGV) and then receive daily subcutaneous vosoritide (15 mcg/kg/day) for 12 months.
Report a problemFND-9237898701
02HumanNon-randomized trialEfficacy outcome
In two children with NPR2 mutations treated with vosoritide, annualized growth velocities were increased, at 4.4 and 9.2 cm/yr.
In plain wordsTwo children with NPR2 mutations had increased growth velocities of 4.4 and 9.2 cm/yr on vosoritide.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity in NPR2 mutations
Study
n = 2
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15 mcg/kg/day · subcutaneousAs given in this study (human).
Measurements
Annualized growth velocity
4.4 cm/yr
NPR2 mutation, subject 1
Annualized growth velocity
9.2 cm/yr
NPR2 mutation, subject 2
Source sentences
Summary
The two subjects with NPR2 mutations had increased AGVs of 4.4 and 9.2 cm/yr.
1 context sentence(design, population, supporting numbers)
Summary · context
Subjects are followed for a 6-month observation period to establish a baseline annualized growth velocity (AGV) and then receive daily subcutaneous vosoritide (15 mcg/kg/day) for 12 months.
Report a problemFND-9237898702
03HumanNon-randomized trialEfficacy outcome
In two children with Noonan syndrome, vosoritide increased annualized growth velocity by 7.5 and 3.3 cm/yr from baseline.
In plain wordsIn two children with Noonan syndrome, vosoritide increased annualized growth velocity by 7.5 and 3.3 cm/yr from baseline.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity in Noonan syndrome
Study
n = 2
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15 mcg/kg/day · subcutaneousAs given in this study (human).
Measurements
Increase in annualized growth velocity
7.5 cm/yr
Noonan syndrome, subject 1
Increase in annualized growth velocity
3.3 cm/yr
Noonan syndrome, subject 2
Source sentences
Summary
The two subjects with Noonan syndrome had an increase in AGV of 7.5 and 3.3 cm/yr.
1 context sentence(design, population, supporting numbers)
Summary · context
Subjects are followed for a 6-month observation period to establish a baseline annualized growth velocity (AGV) and then receive daily subcutaneous vosoritide (15 mcg/kg/day) for 12 months.
Report a problemFND-9237898703
04HumanNon-randomized trialEfficacy outcome
In one child with Noonan syndrome previously treated with growth hormone, annualized growth velocity was 11.6 cm/year during the first 6 months of vosoritide versus 4.1 cm/yr during the 6-month observation period.
In plain wordsOne child with Noonan syndrome grew at 11.6 cm/year in the first 6 months of vosoritide, versus 4.1 cm/yr in the 6-month observation period.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity in previously GH-treated child
Study
n = 1
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15 mcg/kg/day · subcutaneous · 6 monthsAs given in this study (human).
Measurements
Annualized growth velocity
11.6 cm/year
First 6 months of vosoritide
Annualized growth velocity
4.1 cm/yr
6-month observation period
Source sentences
Summary
His AGV was 6.7 cm/yr while on GH, 4.1 cm/yr during the 6-month observation period, and 11.6 cm/year during the first 6 months of vosoritide treatment.
2 context sentences(design, population, supporting numbers)
Summary · context
One subject with Noonan syndrome, a 4-year-old male, had previously been treated with growth hormone (GH) for 2 years.
Summary · context
Subjects are followed for a 6-month observation period to establish a baseline annualized growth velocity (AGV) and then receive daily subcutaneous vosoritide (15 mcg/kg/day) for 12 months.
Report a problemFND-9237898704
05HumanNon-randomized trialEfficacy outcome
In one child with Noonan syndrome, annualized growth velocity was 11.6 cm/year during the first 6 months of vosoritide versus 6.7 cm/yr while on growth hormone.
In plain wordsOne child with Noonan syndrome grew at 11.6 cm/year in the first 6 months of vosoritide, versus 6.7 cm/yr on growth hormone.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity versus prior GH therapy
Study
n = 1
Comparator
Prior growth hormone treatment
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15 mcg/kg/day · subcutaneous · 6 monthsAs given in this study (human).
Measurements
Annualized growth velocity
11.6 cm/year
First 6 months of vosoritide
Annualized growth velocity
6.7 cm/yr
While on growth hormone
Source sentences
Summary
His AGV was 6.7 cm/yr while on GH, 4.1 cm/yr during the 6-month observation period, and 11.6 cm/year during the first 6 months of vosoritide treatment.
2 context sentences(design, population, supporting numbers)
Summary · context
One subject with Noonan syndrome, a 4-year-old male, had previously been treated with growth hormone (GH) for 2 years.
Summary · context
Subjects are followed for a 6-month observation period to establish a baseline annualized growth velocity (AGV) and then receive daily subcutaneous vosoritide (15 mcg/kg/day) for 12 months.
Report a problemFND-9237898705
06HumanNon-randomized trialSafety outcome
In children with genetic short stature receiving vosoritide, no treatment-related serious adverse events occurred.
In plain wordsIn children with genetic short stature receiving vosoritide, no treatment-related serious adverse events occurred.
Peptide
Vosoritide
Outcome
Not applicableTreatment-related serious adverse events
Study
n: not reported in source
Outcome group
Adverse events · Whole body
Studied dose
15 mcg/kg/day · subcutaneousAs given in this study (human).
Source sentences
Summary
There were no serious adverse events related to vosoritide treatment.
1 context sentence(design, population, supporting numbers)
Summary · context
Subjects are followed for a 6-month observation period to establish a baseline annualized growth velocity (AGV) and then receive daily subcutaneous vosoritide (15 mcg/kg/day) for 12 months.
Report a problemFND-9237898706
07HumanNon-randomized trialSafety outcome
One child had brief, self-resolving syncope with her first vosoritide injection and continued therapy without further incidents.
In plain wordsOne child had brief, self-resolving syncope with her first vosoritide injection and continued therapy without further incidents.
Peptide
Vosoritide
Outcome
Not applicableSyncope
Study
n: not reported in source
Outcome group
Adverse events · Cardiovascular
Studied dose
15 mcg/kg/day · subcutaneousAs given in this study (human).
Source sentences
Summary
One subject had a brief episode of syncope with her first injection which self-resolved, and she continued on therapy with no further incidents.
1 context sentence(design, population, supporting numbers)
Summary · context
Subjects are followed for a 6-month observation period to establish a baseline annualized growth velocity (AGV) and then receive daily subcutaneous vosoritide (15 mcg/kg/day) for 12 months.
Report a problemFND-9237898707
08HumanNon-randomized trialPharmacokinetics
In children with genetic short stature, vosoritide pharmacokinetic parameters resembled published parameters in children with achondroplasia.
In plain wordsIn children with genetic short stature, vosoritide pharmacokinetic parameters resembled published parameters in children with achondroplasia.
Peptide
Vosoritide
Outcome
Direction not reportedPharmacokinetic parameters
Study
n: not reported in source
Comparator
Previously published parameters in children with achondroplasia
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
15 mcg/kg/day · subcutaneousAs given in this study (human).
Source sentences
Summary
PK parameters were similar to previously published parameters for vosoritide in children with achondroplasia.
1 context sentence(design, population, supporting numbers)
Summary · context
Subjects are followed for a 6-month observation period to establish a baseline annualized growth velocity (AGV) and then receive daily subcutaneous vosoritide (15 mcg/kg/day) for 12 months.
Report a problemFND-9237898708
09HumanObservational studyActivity measurement
In preliminary data from children receiving vosoritide, Cmax correlated strongly with peak cGMP increase (r=0.70, p=0.01).
In plain wordsIn preliminary data from children receiving vosoritide, higher peak drug concentrations were linked to greater peak cGMP increases.
Peptide
Vosoritide
Outcome
IncreaseCmax association with peak cGMP increase
Study
n: not reported in source
Outcome group
Receptor activity and mechanism · Pharmacology
Studied dose
15 mcg/kg/day · subcutaneousAs given in this study (human).
Measurements
Correlation of Cmax with peak cGMP increase
0.7 r
Preliminary data
P value
0.01 p
Cmax versus peak cGMP increase
Source sentences
Summary
In preliminary data, vosoritide Cmax and AUC correlated strongly with peak increase in cGMP (r=0.70, p=0.01;r=0.84, p<0.01, respectively) but not with increase in AGV (r=0.35, p=0.32).
1 context sentence(design, population, supporting numbers)
Summary · context
Subjects are followed for a 6-month observation period to establish a baseline annualized growth velocity (AGV) and then receive daily subcutaneous vosoritide (15 mcg/kg/day) for 12 months.
Report a problemFND-9237898709
10HumanObservational studyActivity measurement
In preliminary data from children receiving vosoritide, AUC correlated strongly with peak cGMP increase (r=0.84, p<0.01).
In plain wordsIn preliminary data from children receiving vosoritide, greater total drug exposure was linked to greater peak cGMP increases.
Peptide
Vosoritide
Outcome
IncreaseAUC association with peak cGMP increase
Study
n: not reported in source
Outcome group
Receptor activity and mechanism · Pharmacology
Studied dose
15 mcg/kg/day · subcutaneousAs given in this study (human).
Measurements
Correlation of AUC with peak cGMP increase
0.84 r
Preliminary data
P value
< 0.01 p
AUC versus peak cGMP increase
Source sentences
Summary
In preliminary data, vosoritide Cmax and AUC correlated strongly with peak increase in cGMP (r=0.70, p=0.01;r=0.84, p<0.01, respectively) but not with increase in AGV (r=0.35, p=0.32).
1 context sentence(design, population, supporting numbers)
Summary · context
Subjects are followed for a 6-month observation period to establish a baseline annualized growth velocity (AGV) and then receive daily subcutaneous vosoritide (15 mcg/kg/day) for 12 months.
Report a problemFND-9237898710
11HumanObservational studyEfficacy outcome
In preliminary data from children receiving vosoritide, exposure did not significantly correlate with growth velocity increase despite a positive coefficient (r=0.35, p=0.32).
In plain wordsIn preliminary data from children receiving vosoritide, the positive link between drug exposure and growth velocity increase was not statistically significant.
Peptide
Vosoritide
Outcome
IncreaseExposure association with growth velocity increase
Study
n: not reported in source
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15 mcg/kg/day · subcutaneousAs given in this study (human).
Measurements
Correlation of exposure with increase in annualized growth velocity
0.35 r
Preliminary data
P value
0.32 p
Exposure versus AGV increase
Source sentences
Summary
In preliminary data, vosoritide Cmax and AUC correlated strongly with peak increase in cGMP (r=0.70, p=0.01;r=0.84, p<0.01, respectively) but not with increase in AGV (r=0.35, p=0.32).
1 context sentence(design, population, supporting numbers)
Summary · context
Subjects are followed for a 6-month observation period to establish a baseline annualized growth velocity (AGV) and then receive daily subcutaneous vosoritide (15 mcg/kg/day) for 12 months.
Report a problemFND-9237898711

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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.

Publication types

Abstract

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