HypochondroplasiaPhase 3CANOPY-HCH-3NCT06455059
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
A Phase 3 study testing Vosoritide in people with Hypochondroplasia, run by BioMarin Pharmaceutical at 23 sites in 9 countries.
Trial 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 more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Hypochondroplasia
- What is being tested?
- Vosoritide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 23 sitesin 9 countries
- Study dates
- 17 Jun 2024to 1 Aug 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participants must be ≥ 3 to < 18 years of age at enrollment
- A confirmed genetic diagnosis of HCH
- 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
- Males and females are eligible to participate in this clinical study.
- 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.
- If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.
Who cannot join
- Short stature condition other than HCH
- Have an unstable condition likely to require surgical intervention during the study.
- Evidence of decreased growth velocity and/or growth plate closure
- Taking any of the prohibited medications
- Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids
- Planned or expected to have limb-lengthening surgery during the study period.
Show all 13Show fewer
- Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period
- Require any investigational agent prior to completion of study period.
- Received vosoritide or another investigational product or investigational medical device in the past
- Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide
- Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
- Have known hypersensitivity to vosoritide or its excipients.
- Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.
Trial locations and contacts
23 sites in 9 countries.
23 sites in 9 countries
United States4 sites
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Nemours Alfred I. DuPont Hospital for Children
Wilmington, Delaware, 19803
France4 sites
Hospices Civils de Lyon - Hopital Femme Mere Enfant
Lyon
Hopital de la Timone
Marseille
Hopital Necker-Enfants Malade
Paris
CHU de Toulouse
Toulouse
Japan4 sites
Osaka University Hosptial
Osaka
Tokushima University Hospital
Tokushima
Institute of Science Tokyo Hospital
Tokyo
Tottori University Hospital
Tottori
Canada3 sites
University of Alberta Stollery Children's Hospital
Edmonton, Alberta
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec
SickKids - The Hospital for Sick Children
Toronto, Ontario
Italy3 sites
Instituto Giannina Gaslini
Genoa
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome
Ospedale Pediatrico Bambino Gesu
Rome
Germany2 sites
Uniklinik Koln
Cologne
Univeristatskinderklinik Magdeburg
Magdeburg
Australia1 site
Murdoch Children's Research Institute
Parkville, Victoria, 3052
United Kingdom1 site
Great Ormond Street Hospital for Children NHS Foundation Trust
London
About this study
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children With Hypochondroplasia
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.
- Conditions
- Hypochondroplasia
- Being tested
- Vosoritide, Placebo
- 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
- Medical subject headings
- Hypochondroplasia, Dwarfism, Bone Diseases, Bone Diseases, Developmental, Musculoskeletal Diseases, Genetic Diseases, Inborn, Osteochondrodysplasias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VosoritideDrug
Also called Modified recombinant human C-type natriuretic peptide
Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
PlaceboDrug
Subcutaneous injection of recommended dose of placebo
| Study group | Role | What they receive |
|---|---|---|
| vosoritide injection with vial and syringe | Experimental | Not described Receives: Drug: Vosoritide |
| Placebo injection with vial and syringe | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo
Measured: At week 52
Secondary outcomes (2)
Change from baseline in standing height at Week 52 versus placebo
Measured: At week 52
Change from baseline in height Z-score at Week 52 versus placebo
Measured: At week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2024
- Study start17 Jun 2024
- Primary completion1 Aug 2026 (estimated)
- Study completion1 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HypochondroplasiaStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06455059
- Sponsor's protocol ID
- 111-303
- Acronym
- CANOPY-HCH-3
- Status last verified
- Jan 2026
- First submitted
- 7 Jun 2024
- Last updated
- 20 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Dauber A, Savarirayan R, Campeau PM, Carroll RS, Cheung M, Cormier-Daire V, de Bergua JM, Edouard T, Gonfiantini MV, Hoover-Fong J, Kannu P, Kinoshita Y, Kubota T, Mendoza-Londono R, Namba N, Ohata Y, Onesimo R, Palm K, Prada CE, Reynaud R, Rossi M, Semler O, Takasawa K, Bassi R, Ghani S, Huntsman Labed A, Osmond D, Sabir I, White J, Maghnie M. A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia. NEJM Evid. 2026 Sep 9:EVIDoa2600257. doi: 10.1056/EVIDoa2600257. Online ahead of print.
PubMed 42713641
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children With Hypochondroplasia
- ClinicalTrials.gov ID
- NCT06455059
- Acronym / sponsor ID
- CANOPY-HCH-3 / 111-303
- Conditions
- Hypochondroplasia
- Keywords
- Hypochondroplasia, Dwarfism, Bone Diseases, Bone Disease, Developmental, HCH, Natriuretic Peptide, C-Type, Musculoskeletal Diseases, Natriuretic Agents
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 17 Jun 2024 (actual)
- Primary completion
- 1 Aug 2026 (estimated)
- Study completion
- 1 Aug 2026 (estimated)
- First posted
- 12 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| vosoritide injection with vial and syringe | Experimental | Drug: Vosoritide |
| Placebo injection with vial and syringe | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Participants must be ≥ 3 to < 18 years of age at enrollment
- A confirmed genetic diagnosis of HCH
- 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
- Males and females are eligible to participate in this clinical study.
- 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.
Key exclusion criteria (13 listed)
- Short stature condition other than HCH
- Have an unstable condition likely to require surgical intervention during the study.
- Evidence of decreased growth velocity and/or growth plate closure
- Taking any of the prohibited medications
- Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VosoritideAlso: Modified recombinant human C-type natriuretic peptideSubcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily. | Drug | vosoritide injection with vial and syringe |
| PlaceboSubcutaneous injection of recommended dose of placebo | Drug | Placebo injection with vial and syringe |
Primary outcome
Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo
Time frame: At week 52
2 secondary outcomes
Change from baseline in standing height at Week 52 versus placebo
Time frame: At week 52
Change from baseline in height Z-score at Week 52 versus placebo
Time frame: At week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2026.
- Derived from this study: PubMed 42713641Dauber A, Savarirayan R, Campeau PM, Carroll RS, Cheung M, Cormier-Daire V, de Bergua JM, Edouard T, Gonfiantini MV, Hoover-Fong J, Kannu P, Kinoshita Y, Kubota T, Mendoza-Londono R, Namba N, Ohata Y, Onesimo R, Palm K, Prada CE, Reynaud R, Rossi M, Semler O, Takasawa K, Bassi R, Ghani S, Huntsman Labed A, Osmond D, Sabir I, White J, Maghnie M. A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia. NEJM Evid. 2026 Sep 9:EVIDoa2600257. doi: 10.1056/EVIDoa2600257. Online ahead of print.
- Individual participant dataSharing planned: No
Related trials for Hypochondroplasia
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director, MD study director
BioMarin Pharmaceutical
23 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Vosoritide in people with Hypochondroplasia, run by BioMarin Pharmaceutical at 23 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 20 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, France, Japan, Canada, Italy and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo (time frame: At week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06455059, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2026.