HypochondroplasiaPhase 3CANOPY-HCH-EXTNCT07073014
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
A Phase 3 study testing Vosoritide in people with Hypochondroplasia, run by BioMarin Pharmaceutical at 30 sites in 9 countries.
Trial summary
The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hypochondroplasia
- What is being tested?
- Vosoritide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 140 participants
- Where
- 30 sitesin 9 countries
- Study dates
- 20 Jun 2025to Oct 2044
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants must have completed the Week 52 visit for 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays
- Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any study-related procedure
- Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Baseline 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
- Participants are willing and able to perform all study procedures as physically possible
- Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training
Who cannot join
- Permanently discontinued study treatment in the studies 111-303 or 111-212
- Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care
- Taking or planning to take any prohibited medications
- Planned or expected to have limb-lengthening surgery during the study period
- Planned or expected bone-related surgery during the study period
- Require any investigational agent prior to completion of study period
Show all 10Show fewer
- Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy
- Have known hypersensitivity to vosoritide or its excipients
- Is pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study
- 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
30 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
30 sites in 9 countries
United States10 sites
The Johns Hopkins University School of Medicine
Baltimore, Maryland, 21205
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
University of Missouri
Columbia, Missouri, 65201
Cook Children's Endocrinology
Fort Worth, Texas, 76104
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, 55454
Benioff Children's Hospital - Oakland
Oakland, California, 94609
Phoenix Children's Hospital - Thomas Campus
Phoenix, Arizona, 85016
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Nemours Children's Hospital, Delaware (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
Germany3 sites
Uniklinik Koln
Cologne
Universitätsklinikum des Saarlandes
Homburg, Saarland, 66421
Univeristatskinderklinik Magdeburg
Magdeburg
Italy3 sites
Instituto Giannina Gaslini
Genoa
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome
Ospedale Pediatrico Bambino Gesu
Rome
Australia1 site
Murdoch Children's Research Institute of The Royal Children's Hospital
Parkville, Victoria, 3052
United Kingdom1 site
Great Ormond Street Hospital for Children NHS Foundation Trust
London
About this study
Official title: A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide in Children With Hypochondroplasia
The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
- Conditions
- Hypochondroplasia
- Being tested
- Vosoritide
- Medical subject headings
- Hypochondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VosoritideDrug
Open-label administration of vosoritide using weight-band dosing
| Study group | Role | What they receive |
|---|---|---|
| Vosoritide | Experimental | Open-label active drug Receives: Drug: Vosoritide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)
As measured by change from baseline in height Z-score
Measured: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Secondary outcomes (11)
Evaluate the maintenance effect of vosoritide treatment on standing height
change from baseline in height at Week 52
Measured: Baseline to week 52
Evaluate the maintenance effect of vosoritide treatment on standing height Z-score
Change from baseline in height Z-score at Week 52
Measured: Baseline to week 52
Evaluate long-term effect of vosoritide treatment on growth velocity
Absolute 6- or 12-month interval AGV
Measured: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on body proportionality
Change from baseline in upper to lower body segment ratio
Measured: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on bone age versus chronological age
As measured by left-hand and wrist x-ray
Measured: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on bone metabolism biomarkers
Change from baseline in serum collagen X marker
Measured: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term changes in Health-Related Quality of Life (HRQoL)
Change from baseline as measured by Quality of Life In Short-Statured Youth (QoLISSY) total score
Measured: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on bone quality
Change from baseline in bone mineral density (BMD) as measured by Dual X-Ray Absorptiometry
Measured: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on pubertal growth as measured by Tanner Staging
Measured: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
To monitor the incidence of otitis media
Measured: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
To monitor the frequency of seizures
Measured: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2025
- Study start20 Jun 2025
- Primary completionOct 2044 (estimated)
- Study completionOct 2044 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 140 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07073014
- Sponsor's protocol ID
- 111-308
- Acronym
- CANOPY-HCH-EXT
- Status last verified
- Sep 2026
- First submitted
- 27 Jun 2025
- Last updated
- 9 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide in Children With Hypochondroplasia
- ClinicalTrials.gov ID
- NCT07073014
- Acronym / sponsor ID
- CANOPY-HCH-EXT / 111-308
- Conditions
- Hypochondroplasia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 140 participants (estimated)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 20 Jun 2025 (actual)
- Primary completion
- Oct 2044 (estimated)
- Study completion
- Oct 2044 (estimated)
- First posted
- 18 Jul 2025 (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 purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vosoritide | Experimental | Drug: VosoritideOpen-label active drug |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Participants must have completed the Week 52 visit for 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays
- Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any study-related procedure
- Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Baseline 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
- Participants are willing and able to perform all study procedures as physically possible
Key exclusion criteria (10 listed)
- Permanently discontinued study treatment in the studies 111-303 or 111-212
- Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care
- Taking or planning to take any prohibited medications
- Planned or expected to have limb-lengthening surgery during the study period
- Planned or expected bone-related surgery during the study period
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 |
|---|---|---|
| VosoritideOpen-label administration of vosoritide using weight-band dosing | Drug | Vosoritide |
Primary outcome
Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
As measured by change from baseline in height Z-score
11 secondary outcomes
Evaluate the maintenance effect of vosoritide treatment on standing height
Time frame: Baseline to week 52
Evaluate the maintenance effect of vosoritide treatment on standing height Z-score
Time frame: Baseline to week 52
Evaluate long-term effect of vosoritide treatment on growth velocity
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on body proportionality
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on bone age versus chronological age
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on bone metabolism biomarkers
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term changes in Health-Related Quality of Life (HRQoL)
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on bone quality
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on pubertal growth as measured by Tanner Staging
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
To monitor the incidence of otitis media
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
To monitor the frequency of seizures
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
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 9 Sep 2026.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
30 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 30 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 10 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: enrolling by invitation, as of the sponsor's last update on 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in United States, France, Japan, Canada, Germany 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. 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?
Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH) (time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Sep 2026.