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Active, not recruitingNCT06455059CANOPY-HCH-3Updated Jan 20, 2026

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

A Phase 3 interventional study of Vosoritide and Placebo in Hypochondroplasia, sponsored by BioMarin Pharmaceutical. Active, not recruiting at 23 sites in 9 countries. Open to participants aged 3 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-01-20.

Sponsored by BioMarin Pharmaceutical · Phase 3, Interventional, and Treatment

From the registry’s dates

  • Primary completion was expected by Aug 2026, 3 months ago, but the record still lists the study as active, not recruiting.
Phase
Phase 3
Study type
Interventional
Enrollment
80
Allocation
Randomized
Ages
3 Years to 17 Years
Sex
All
01

Study summary

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).

Read the detailed description

This is a Phase 3 randomized, stratified, placebo-controlled, double-blind multicenter study to evaluate the effect of 52 weeks of daily vosoritide administration on annualized growth velocity (AGV) in participants with HCH. Eligible participants with documented HCH confirmed by genetic testing will roll over from Study 111-902 and enter the 111-303 study. Participants will be randomly assigned to one of two treatment groups: Placebo or Vosoritide. The route of administration is subcutaneous injection, and the frequency of administration is daily.

02

Conditions studied

  • Hypochondroplasia

Keywords

  • Hypochondroplasia
  • Dwarfism
  • Bone Diseases
  • Bone Disease, Developmental
  • HCH
  • Natriuretic Peptide, C-Type
  • Musculoskeletal Diseases
  • Natriuretic Agents
  • Physiological Effects of Drugs
  • Skeletal Dysplasias
  • Genetic Diseases, Inborn
  • Osteochondrodysplasias
  • Vosoritide
  • Voxzogo
03

In context

Dwarfism

99 studies on the registry are indexed under Dwarfism; 12 are open to participants now.

This study's planned enrollment of 80 is above the median of 60 across 66 interventional studies indexed under Dwarfism.

Browse Dwarfism studies →

Lead sponsor

BioMarin Pharmaceutical is the lead sponsor of 112 studies on the registry; 13 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.

Browse BioMarin Pharmaceutical studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
3 Years to 17 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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 while participating in the study.

Exclusion criteria

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 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 , history of malignancy, or currently under work-up for suspected malignancy.
  12. Have known to vosoritide or its .
  13. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.
Terms in these criteria, in plain language(5)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

Excipients

The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Single group
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
80 participants (estimated)

Study arms

  • Experimental
    vosoritide injection with vial and syringe

    Drug: Vosoritide

  • Placebo comparator
    Placebo injection with vial and syringe

    Drug: Placebo

Interventions

  • DrugVosoritide

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

    Also known as: Modified recombinant human C-type natriuretic peptide

  • DrugPlacebo

    Subcutaneous injection of recommended dose of placebo

06

What researchers measure

Primary outcomes

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

    Time frame: At week 52

Secondary outcomes

  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

07

Study locations

23 sites
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Jun 2024 (actual). Primary completion is estimated for Aug 2026, 25 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Jun 7, 2024
First posted
Jun 12, 2024
StartActual
Jun 17, 2024
Primary completionEst.
Aug 1, 2026
CompletionEst.
Aug 1, 2026
Last update
Jan 20, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jan 20, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06455059
Lead sponsor
BioMarin Pharmaceutical
Responsible party
Sponsor
First posted
Jun 12, 2024
Start date
Jun 17, 2024
Primary completion
Aug 1, 2026 (estimated)
Completion
Aug 1, 2026 (estimated)
Last update
Jan 20, 2026

Study contacts

Medical Director, MD
study director · BioMarin Pharmaceutical

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Jan 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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