Phase 2 Trial of Vosoritide Use in Patients with Hypochondroplasia: A Pharmacokinetic/Pharmacodynamic Analysis.

Galetaki D, Zhang A, Qi Y, Merchant N, Kanakatti Shankar R, Boucher K, Shafaei N, Seaforth R, Dham N, Dauber A

Hormone research in paediatrics18 Oct 2024

PMID39427650DOI10.1159/000542102PepBankPUB-39427650

Publisher page PubMedAsk about this study

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 study9

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

01HumanNon-randomized trialMechanism
In children with hypochondroplasia, CXM increased from 22.5 ± 6.5 to 41.6 ± 15.9 ng/mL after 12 months of vosoritide (p < 0.0001).
In plain wordsIn children with hypochondroplasia, vosoritide increased the collagen X biomarker versus baseline.
Peptide
Vosoritide
Outcome
IncreaseCollagen X biomarker
Study
n = 24
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15 μg/kg/day · subcutaneous · 12 monthsAs given in this study (human).
Measurements
CXM baseline mean
22.5 ng/mL
baseline
CXM baseline spread (±)
6.5 ng/mL
baseline
CXM mean
41.6 ng/mL
after 12 months of treatment
CXM spread (±)
15.9 ng/mL
after 12 months of treatment
P
< 0.0001
12 months versus baseline
Source sentences
Summary
CXM levels increased from a baseline mean of 22.5 ± 6.5 to 41.6 ± 15.9 ng/mL after 12 months of treatment (p < 0.0001).
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a phase II trial of daily subcutaneous vosoritide (15 μg/kg/day) in 24 prepubertal subjects with hypochondroplasia (12 females, mean age 5.9 ± 2.3 years, mean height -3.29 + 0.68 SD).
Summary · context
PD markers including serum collagen X biomarker (CXM) and urine cyclic guanosine monophosphate (cGMP) production were measured at day 1, month 6, and month 12 visits.
Report a problemFND-3942765002
02HumanNon-randomized trialMechanism
In children with hypochondroplasia, urine cGMP increased within 1 h and peaked at 2 h after vosoritide injection.
In plain wordsIn children with hypochondroplasia, urine cGMP increased after vosoritide injection and peaked at two hours.
Peptide
Vosoritide
Outcome
IncreaseUrine cGMP response
Study
n = 24
Outcome group
Receptor activity and mechanism · Pharmacology
Studied dose
15 μg/kg/day · subcutaneousAs given in this study (human).
Measurements
Urine cGMP increase onset
≤ 1 h
after injection
Time to peak urine cGMP
2 h
after injection
Source sentences
Summary
Urine cGMP increased within 1 h and peaked at 2 h after injection.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a phase II trial of daily subcutaneous vosoritide (15 μg/kg/day) in 24 prepubertal subjects with hypochondroplasia (12 females, mean age 5.9 ± 2.3 years, mean height -3.29 + 0.68 SD).
Summary · context
PD markers including serum collagen X biomarker (CXM) and urine cyclic guanosine monophosphate (cGMP) production were measured at day 1, month 6, and month 12 visits.
Report a problemFND-3942765003
03HumanNon-randomized trialMechanism
In children receiving vosoritide for hypochondroplasia, mean cGMP production AUC did not differ significantly across study visits.
In plain wordsIn children receiving vosoritide, average cGMP production did not differ significantly between study visits.
Peptide
Vosoritide
Outcome
No significant changecGMP AUC across visits
Study
n = 24
Comparator
Other study visits in the same participants
Outcome group
Receptor activity and mechanism · Pharmacology
Studied dose
15 μg/kg/day · subcutaneousAs given in this study (human).
Source sentences
Summary
The mean AUC for cGMP production was not significantly different at each study visit.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a phase II trial of daily subcutaneous vosoritide (15 μg/kg/day) in 24 prepubertal subjects with hypochondroplasia (12 females, mean age 5.9 ± 2.3 years, mean height -3.29 + 0.68 SD).
Summary · context
PD markers including serum collagen X biomarker (CXM) and urine cyclic guanosine monophosphate (cGMP) production were measured at day 1, month 6, and month 12 visits.
Report a problemFND-3942765004
04HumanNon-randomized trialPharmacokinetics
In children with hypochondroplasia, vosoritide PK parameters were similar to previously reported parameters in achondroplasia.
In plain wordsIn children with hypochondroplasia, vosoritide handling was similar to previous reports in achondroplasia.
Peptide
Vosoritide
Outcome
Not applicablePK parameters
Study
n: not reported in source
Comparator
Previously reported achondroplasia patients
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
15 μg/kg/day · subcutaneousAs given in this study (human).
Source sentences
Summary
Vosoritide PK parameters were similar to those previously reported in patients with achondroplasia.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a phase II trial of daily subcutaneous vosoritide (15 μg/kg/day) in 24 prepubertal subjects with hypochondroplasia (12 females, mean age 5.9 ± 2.3 years, mean height -3.29 + 0.68 SD).
Summary · context
PD markers including serum collagen X biomarker (CXM) and urine cyclic guanosine monophosphate (cGMP) production were measured at day 1, month 6, and month 12 visits.
Report a problemFND-3942765001
05HumanObservational studyMechanism
In children receiving vosoritide for hypochondroplasia, maximum cGMP AUC change correlated with PK AUC (r = 0.46, p = 0.0001).
In plain wordsIn children receiving vosoritide, greater drug exposure was linked to a greater maximum cGMP response.
Peptide
Vosoritide
Outcome
IncreasecGMP AUC and PK AUC correlation
Study
n = 24
Outcome group
Receptor activity and mechanism · Pharmacology
Studied dose
15 μg/kg/day · subcutaneousAs given in this study (human).
Measurements
correlation coefficient (r)
0.46
maximum change in cGMP AUC versus PK AUC
p-value
0.0001
maximum change in cGMP AUC versus PK AUC
Source sentences
Summary
The maximum change in cGMP AUC correlated with PK AUC (r = 0.46, p = 0.0001).
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a phase II trial of daily subcutaneous vosoritide (15 μg/kg/day) in 24 prepubertal subjects with hypochondroplasia (12 females, mean age 5.9 ± 2.3 years, mean height -3.29 + 0.68 SD).
Summary · context
PD markers including serum collagen X biomarker (CXM) and urine cyclic guanosine monophosphate (cGMP) production were measured at day 1, month 6, and month 12 visits.
Report a problemFND-3942765005
06HumanObservational studyMechanism
In children receiving vosoritide for hypochondroplasia, average PK AUC did not correlate with any growth outcome.
In plain wordsIn children receiving vosoritide, average drug exposure was not linked to growth outcomes.
Peptide
Vosoritide
Outcome
No significant changeDrug exposure and growth outcomes
Study
n = 24
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15 μg/kg/day · subcutaneousAs given in this study (human).
Source sentences
Summary
However, drug exposure, as measured by average PK AUC, did not correlate with any growth outcome.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a phase II trial of daily subcutaneous vosoritide (15 μg/kg/day) in 24 prepubertal subjects with hypochondroplasia (12 females, mean age 5.9 ± 2.3 years, mean height -3.29 + 0.68 SD).
Summary · context
PD markers including serum collagen X biomarker (CXM) and urine cyclic guanosine monophosphate (cGMP) production were measured at day 1, month 6, and month 12 visits.
Report a problemFND-3942765006
07HumanObservational studyMechanism
In children receiving vosoritide, CXM correlated with prior 6-month height velocity (partial correlation coefficient = 0.40, p = 0.0048).
In plain wordsIn children receiving vosoritide, higher CXM levels were linked to faster height growth during the prior interval.
Peptide
Vosoritide
Outcome
IncreaseCXM and prior height velocity correlation
Study
n = 24
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15 μg/kg/day · subcutaneousAs given in this study (human).
Measurements
partial correlation coefficient
0.4
CXM versus prior 6-month interval height velocity
p-value
0.0048
CXM versus prior 6-month interval height velocity
Source sentences
Summary
CXM levels correlated with the prior 6-month interval height velocity (partial correlation coefficient = 0.40, p = 0.0048).
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a phase II trial of daily subcutaneous vosoritide (15 μg/kg/day) in 24 prepubertal subjects with hypochondroplasia (12 females, mean age 5.9 ± 2.3 years, mean height -3.29 + 0.68 SD).
Summary · context
PD markers including serum collagen X biomarker (CXM) and urine cyclic guanosine monophosphate (cGMP) production were measured at day 1, month 6, and month 12 visits.
Report a problemFND-3942765007
08HumanObservational studyMechanism
In children receiving vosoritide, CXM change did not correlate with height velocity change during treatment.
In plain wordsIn children receiving vosoritide, changes in CXM were not linked to changes in height growth speed.
Peptide
Vosoritide
Outcome
No significant changeCXM change and height velocity change
Study
n = 24
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15 μg/kg/day · subcutaneousAs given in this study (human).
Source sentences
Summary
However, change in CXM did not correlate with change in height velocity or change in height SD during treatment.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a phase II trial of daily subcutaneous vosoritide (15 μg/kg/day) in 24 prepubertal subjects with hypochondroplasia (12 females, mean age 5.9 ± 2.3 years, mean height -3.29 + 0.68 SD).
Summary · context
PD markers including serum collagen X biomarker (CXM) and urine cyclic guanosine monophosphate (cGMP) production were measured at day 1, month 6, and month 12 visits.
Report a problemFND-3942765008
09HumanObservational studyMechanism
In children receiving vosoritide, CXM change did not correlate with height SD change during treatment.
In plain wordsIn children receiving vosoritide, changes in CXM were not linked to changes in height SD.
Peptide
Vosoritide
Outcome
No significant changeCXM change and height SD change
Study
n = 24
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15 μg/kg/day · subcutaneousAs given in this study (human).
Source sentences
Summary
However, change in CXM did not correlate with change in height velocity or change in height SD during treatment.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a phase II trial of daily subcutaneous vosoritide (15 μg/kg/day) in 24 prepubertal subjects with hypochondroplasia (12 females, mean age 5.9 ± 2.3 years, mean height -3.29 + 0.68 SD).
Summary · context
PD markers including serum collagen X biomarker (CXM) and urine cyclic guanosine monophosphate (cGMP) production were measured at day 1, month 6, and month 12 visits.
Report a problemFND-3942765009

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

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

MeSH terms

Keywords

PepBank is a research reference. Talk to a doctor before you use any peptide.