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Trial · Interventional

Active, not recruitingPhase 3Registry data

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

Short name CANOPY-HCH-3

Overview

Status
Active, not recruiting
Phase
Phase 3
Enrollment
80 participants (estimated)
Sponsor
BioMarin Pharmaceutical · Industry
Design
Randomized · Quadruple masking
Countries
9 countries

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

Start
17 Jun 2024
Primary completion
1 Aug 2026 (estimated)
Completion
1 Aug 2026 (estimated)
First posted
12 Jun 2024
Last update posted
20 Jan 2026
Conditions
Hypochondroplasia

Timeline

1 trial · 1 ongoing

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2024202520262027
  1. Ph 3

    CANOPY-HCH-3

    NCT06455059

    17 Jun 2024 to est. 1 Aug 2026

Registry summary

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

The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).

Interventions

  • VosoritideDrug
  • PlaceboDrug

Arms2

  • Experimental

    vosoritide injection with vial and syringe

  • Placebo comparator

    Placebo injection with vial and syringe

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo

    Time frame · At week 52

Secondary outcomes(2)
  1. Change from baseline in standing height at Week 52 versus placebo

    Time frame · At week 52

  2. Change from baseline in height Z-score at Week 52 versus placebo

    Time frame · At week 52

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

Studies1

Studies that cite this trial.

Studies with results

1 study on this page

PubMed tag: none · 2026

5 results

A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia.

Dauber et al. 2026NEJM evidence

PMID 42713641DOI 10.1056/evidoa2600257PUB-42713641

Full text at the publisher5 results

Eligibility

Who could join, as posted.

Sex
All
Age
from 3 Years up to 17 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: 1. Participants must be ≥ 3 to \< 18 years of age at enrollment 2. A confirmed genetic diagnosis of HCH 3. A height Z score of ≤ - 2.0 standard deviations (SDs) in reference to the general population of the same age and sex, as calculated using the Center for Disease Control and Prevention (CDC) growth charts 4. Males and females are eligible to participate in this clinical study. 5. Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study. 6. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study. Exclusion Criteria: 1. Short stature condition other than HCH 2. Have an unstable condition likely to require surgical intervention during the study. 3. Evidence of decreased growth velocity and/or growth plate closure 4. Taking any of the prohibited medications 5. Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids 6. Planned or expected to have limb-lengthening surgery during the study period. 7. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period 8. Require any investigational agent prior to completion of study period. 9. Received vosoritide or another investigational product or investigational medical device in the past 10. Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide 11. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy. 12. Have known hypersensitivity to vosoritide or its excipients. 13. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.

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