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Trial · Interventional

RecruitingPhase 2Registry data

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

Overview

Status
Recruiting
Phase
Phase 2
Enrollment
60 participants (estimated)
Sponsor
BioMarin Pharmaceutical · Industry
Design
Randomized · Quadruple masking
Countries
7 countries

United States, Australia, France, Germany, Italy, Japan, United Kingdom

Start
30 Jul 2025
Primary completion
30 Jun 2028 (estimated)
Completion
30 Jun 2028 (estimated)
First posted
17 Aug 2025
Last update posted
31 Mar 2026
Conditions
Hypochondroplasia

Timeline

1 trial · 1 ongoing

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20252026202720282029
  1. Ph 2

    A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

    NCT07126262

    30 Jul 2025 to est. 30 Jun 2028

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.

Interventions

  • VosoritideDrug
  • PlaceboDrug

Arms2

  • Experimental

    Vosoritide injection with vial and syringe

    Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.

  • Placebo comparator

    Placebo injection with vial and syringe

    Subcutaneous injection of recommended dose of placebo

Primary outcomes8

Outcomes the sponsor listed as primary.

  1. Incidence of treatment-emergent adverse events

    Time frame · From baseline to end of treatment at 52 weeks

  2. Incidence of serious adverse events versus placebo over the course of the study

    Time frame · From baseline to end of treatment at 52 weeks

  3. Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)

    Time frame · At week 26, at week 52

  4. Changes from baseline in heart rate

    Time frame · At week 13, at week 26, at week 39, at week 52

    Registry description

    Units of measure: bpm

  5. Change from baseline in height Z-score

    Time frame · At week 52

  6. Changes from baseline in respiratory rate

    Time frame · At week 13, at week 26, at week 39, at week 52

    Registry description

    Units of measure: breaths/min

  7. Changes from baseline in temperature

    Time frame · At week 13, at week 26, at week 39, at week 52

    Registry description

    Units of measure: celsius

  8. Changes from baseline in blood pressure

    Time frame · At week 13, at week 26, at week 39, at week 52

    Registry description

    Units of measure: mmHg

Secondary outcomes(19)
  1. Change in height

    Time frame · At week 52

  2. Cumulative annualized growth velocity (AGV)

    Time frame · At week 52

  3. 6-month interval AGV

    Time frame · At week 26, at week 52

  4. Change from baseline in upper to lower body segment ratio

    Time frame · At week 52

  5. Change from baseline in arm span

    Time frame · At week 52

  6. Change from baseline in total body (less head) bone mineral density (BMD) Z-score

    Time frame · At week 52

  7. Change from baseline in lumbar spine BMD Z-score

    Time frame · At week 52

  8. Change from baseline in total body (less head) bone mineral content (BMC) as measured by DXA

    Time frame · At week 52

  9. Change from baseline in lumbar spine BMC as measured by DXA

    Time frame · At week 52

  10. Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)

    Time frame · At week 26, at week 52

  11. Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)

    Time frame · At week 26, at week 52

  12. Elimination half-life of vosoritide (t½)

    Time frame · At week 26, at week 52

  13. Apparent clearance of vosoritide

    Time frame · At week 26, at week 52

  14. Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F)

    Time frame · At week 26, at week 52

  15. Time vosoritide is present at maximum concentration (Tmax)

    Time frame · At week 26, at week 52

  16. Maximum concentration (Cmax) of vosoritide in plasma

    Time frame · At week 26, at week 52

  17. Change from pre-dose at pre-specified timepoints versus placebo in cyclic guanine monophosphate (cGMP)

    Time frame · At week 26 and week 52

  18. Incidence of otitis media

    Time frame · From baseline to end of treatment at 52 weeks

  19. Seizure frequency over the course of the study

    Time frame · From baseline to end of treatment at 52 weeks

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

A trial that is still running, or that stopped early, may post results later.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 0 Months up to 36 Months
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Key Inclusion Criteria: 1. Participants must be 0 to \< 36 months of age at randomization. 2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH). 3. Participants aged 0 to \< 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to \< 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts. 4. Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg. Key Exclusion Criteria: 1. Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia). 2. Have an unstable medical condition likely to require surgical intervention during the study period. 3. Taking any of the prohibited medications. 4. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (\> 3 months) at any time. 5. Require any investigational agent prior to completion of study period. 6. Have received another investigational product or investigational medical device within 30 days prior to the Screening visit. 7. Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time. 8. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy. 9. Have known hypersensitivity to vosoritide or its excipients. 10. Have a condition or circumstance that, in the view of the investigator, places the participant at high risk for poor treatment compliance or for not completing the study. 11. Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason.

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