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Enrolling by invitationNCT07073014CANOPY-HCH-EXTUpdated Sep 9, 2026

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

A Phase 3 interventional study of Vosoritide in Hypochondroplasia, sponsored by BioMarin Pharmaceutical. Enrolling by invitation at 30 sites in 9 countries. Per ClinicalTrials.gov, last updated 2026-09-09.

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

Phase
Phase 3
Study type
Interventional
Enrollment
140
Allocation
Not applicable
Sex
All
01

Study summary

The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH

02

Conditions studied

  • Hypochondroplasia
03

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No

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

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
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
140 participants (estimated)

Study arms

  • Experimental
    Vosoritide

    Open-label active drug

    Drug: Vosoritide

Interventions

  • DrugVosoritide

    Open-label administration of vosoritide using weight-band dosing

05

What researchers measure

Primary outcomes

  1. Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)

    As measured by change from baseline in height Z-score

    Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males

Secondary outcomes

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

    change from baseline in height at Week 52

    Time frame: Baseline to week 52

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

    Change from baseline in height Z-score at Week 52

    Time frame: Baseline to week 52

  3. Evaluate long-term effect of vosoritide treatment on growth velocity

    Absolute 6- or 12-month interval AGV

    Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males

  4. Evaluate long-term effect of vosoritide treatment on body proportionality

    Change from baseline in upper to lower body segment ratio

    Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males

  5. Evaluate long-term effect of vosoritide treatment on bone age versus chronological age

    As measured by left-hand and wrist x-ray

    Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males

  6. Evaluate long-term effect of vosoritide treatment on bone metabolism biomarkers

    Change from baseline in serum collagen X marker

    Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males

  7. 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

    Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males

  8. 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

    Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males

  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

06

Study locations

30 sites
  • Phoenix Children's Hospital - Thomas Campus
    Phoenix, Arizona 85016, United States
  • Benioff Children's Hospital - Oakland
    Oakland, California 94609, United States
  • Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
    Wilmington, Delaware 19803, United States
  • Children's National Medical Center
    Washington D.C., District of Columbia 20010, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago
    Chicago, Illinois 60611, United States
  • The Johns Hopkins University School of Medicine
    Baltimore, Maryland 21205, United States
  • University of Minnesota Masonic Children's Hospital
    Minneapolis, Minnesota 55454, United States
  • University of Missouri
    Columbia, Missouri 65201, United States
  • Cook Children's Endocrinology
    Fort Worth, Texas 76104, United States
  • Medical College of Wisconsin
    Milwaukee, Wisconsin 53226, United States
  • Murdoch Children's Research Institute of The Royal Children's Hospital
    Parkville, Victoria 3052, Australia
  • University of Alberta Stollery Children's Hospital
    Edmonton, Alberta, Canada
  • SickKids - The Hospital for Sick Children
    Toronto, Ontario, Canada
  • Centre Hospitalier Universitaire Sainte-Justine
    Montreal, Quebec, Canada
  • Hospices Civils de Lyon - Hopital Femme Mere Enfant
    Lyon, France
  • Hopital de la Timone
    Marseille, France
  • Hopital Necker-Enfants Malade
    Paris, France
  • CHU de Toulouse
    Toulouse, France
  • Universitätsklinikum des Saarlandes
    Homburg, Saarland 66421, Germany
  • Uniklinik Koln
    Cologne, Germany
  • Univeristatskinderklinik Magdeburg
    Magdeburg, Germany
  • Instituto Giannina Gaslini
    Genoa, Italy
  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
    Rome, Italy
  • Ospedale Pediatrico Bambino Gesu
    Rome, Italy
  • Osaka University Hosptial
    Osaka, Japan
  • Tokushima University Hospital
    Tokushima, Japan
  • Institute of Science Tokyo Hospital
    Tokyo, Japan
  • Tottori University Hospital
    Tottori, Japan
  • Hospital MiKS - Unidad de Cirugía Artroscópica
    Vitoria-Gasteiz, Spain
  • Great Ormond Street Hospital for Children NHS Foundation Trust
    London, United Kingdom
07

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT07073014
Lead sponsor
BioMarin Pharmaceutical
Responsible party
Sponsor
First posted
Jul 18, 2025
Start date
Jun 20, 2025
Primary completion
Oct 2044 (estimated)
Completion
Oct 2044 (estimated)
Last update
Sep 9, 2026

Oversight

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

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