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Enrolling by Invitation

NCT Number: NCT07073014

Long-Term Extension Study of Vosoritide to Treat 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

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Key information

Conditions

Hypochondroplasia

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Murdoch Children's Research Institute, Parkville, Victoria, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Vosoritide

Drug

Open-label administration of vosoritide using weight-band dosing

Primary outcomes

  1. 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

Secondary outcomes

  1. Evaluate the maintenance effect of vosoritide treatment on standing height

    Time frame: Baseline to week 52

    change from baseline in height at Week 52

  2. Evaluate the maintenance effect of vosoritide treatment on standing height Z-score

    Time frame: Baseline to week 52

    Change from baseline in height Z-score at Week 52

  3. 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

    Absolute 6- or 12-month interval AGV

  4. 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

    Change from baseline in upper to lower body segment ratio

  5. 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

    As measured by left-hand and wrist x-ray

  6. 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

    Change from baseline in serum collagen X marker

  7. 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

    Change from baseline as measured by Quality of Life In Short-Statured Youth (QoLISSY) total score

  8. 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

    Change from baseline in bone mineral density (BMD) as measured by Dual X-Ray Absorptiometry

  9. 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

  10. 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

  11. 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

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study title

A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide in Children With Hypochondroplasia

Acronym: CANOPY-HCH-EXT

Important dates

Study start
2025
Primary completion
2040
Study completion
2040
First posted
Jul 18, 2025
Registry last updated
Jul 18, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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