Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia

Short name CANOPY-HCH-EXT

NCTNCT07073014Other id111-308

Open at ClinicalTrials.govAsk about this trial

Overview

Status: Enrolling by invitation
Phase: Phase 3
Enrollment: 140 participants (estimated)
Sponsor: BioMarin Pharmaceutical · Industry
Design: Allocation not applicable · Open label (no masking)
Countries: 9 countries
United States, Australia, Canada, France, Germany, Italy, Japan, Spain, United Kingdom
Start: 20 Jun 2025
Primary completion: Oct 2044 (estimated)
Completion: Oct 2044 (estimated)
First posted: 18 Jul 2025
Last update posted: 9 Sept 2026
Conditions: Hypochondroplasia

Timeline

1 trial · 1 ongoing

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

20252030203520402045

Ph 3
CANOPY-HCH-EXT
NCT07073014
20 Jun 2025 to est. Oct 2044

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 long-term safety and efficacy of daily doses of vosoritide in participants with HCH

Interventions

VosoritideDrug

Arms1

Experimental
Vosoritide
Open-label active drug

Primary outcomes1

Outcomes the sponsor listed as primary.

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
Registry description
As measured by change from baseline in height Z-score

Secondary outcomes(11)

Evaluate the maintenance effect of vosoritide treatment on standing height
Time frame · Baseline to week 52
Registry description
change from baseline in height at Week 52
Evaluate the maintenance effect of vosoritide treatment on standing height Z-score
Time frame · Baseline to week 52
Registry description
Change from baseline in height Z-score at 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
Registry description
Absolute 6- or 12-month interval AGV
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
Registry description
Change from baseline in upper to lower body segment ratio
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
Registry description
As measured by left-hand and wrist x-ray
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
Registry description
Change from baseline in serum collagen X marker
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
Registry description
Change from baseline as measured by Quality of Life In Short-Statured Youth (QoLISSY) total score
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
Registry description
Change from baseline in bone mineral density (BMD) as measured by Dual X-Ray Absorptiometry
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

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: Not stated
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

Inclusion Criteria: * 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 Exclusion Criteria: * 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 * 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

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