Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia (CANOPY-HCH-3)

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).

Study Overview

• Status: Active, not recruiting
• Conditions: Hypochondroplasia
• Intervention / Treatment: Drug: Vosoritide; Drug: Placebo

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 Type: Interventional
• Enrollment (Estimated): 80
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Parkville, Victoria, Australia, 3052
      • Murdoch Children's Research Institute
• Canada
  • Alberta
    • Edmonton, Alberta, Canada
      • University of Alberta Stollery Children's Hospital
  • Ontario
    • Toronto, Ontario, Canada
      • SickKids - The Hospital for Sick Children
  • Quebec
    • Montreal, Quebec, Canada
      • Centre Hospitalier Universitaire Sainte-Justine
• France
  • Lyon, France
    • Hospices Civils de Lyon - Hopital Femme Mère Enfant
  • Marseille, France
    • Hopital de la Timone
  • Paris, France
    • Hopital Necker-Enfants Malade
  • Toulouse, France
    • CHU de Toulouse
• Germany
  • Cologne, Germany
    • Uniklinik Koln
  • Magdeburg, Germany
    • Univeristatskinderklinik Magdeburg
• Italy
  • Genoa, Italy
    • Instituto Giannina Gaslini
  • Rome, Italy
    • Ospedale Pediatrico Bambino Gesu
  • Rome, Italy
    • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
• Japan
  • Osaka, Japan
    • Osaka University Hosptial
  • Tokushima, Japan
    • Tokushima University Hospital
  • Tokyo, Japan
    • Institute of Science Tokyo Hospital
  • Tottori, Japan
    • Tottori University Hospital
• Spain
  • Vitoria-Gasteiz, Spain
    • Vithas Hospital San Jose
• United Kingdom
  • London, United Kingdom
    • Great Ormond Street Hospital for Children NHS foundation trust
• United States
  • Delaware
    • Wilmington, Delaware, United States, 19803
      • Nemours Alfred I. Dupont Hospital for Children
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010
      • Children's National Medical Center
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Medical College of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Participants must be ≥ 3 to < 18 years of age at enrollment
2. A confirmed genetic diagnosis of HCH
3. 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
4. Males and females are eligible to participate in this clinical study.
5. 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.
6. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.

Exclusion Criteria:

1. Short stature condition other than HCH
2. Have an unstable condition likely to require surgical intervention during the study.
3. Evidence of decreased growth velocity and/or growth plate closure
4. Taking any of the prohibited medications
5. Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids
6. Planned or expected to have limb-lengthening surgery during the study period.
7. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period
8. Require any investigational agent prior to completion of study period.
9. Received vosoritide or another investigational product or investigational medical device in the past
10. Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide
11. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
12. Have known hypersensitivity to vosoritide or its excipients.
13. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Single Group Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: vosoritide injection with vial and syringe	Drug: Vosoritide; Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.; Other Names: Modified recombinant human C-type natriuretic peptide
Placebo Comparator: Placebo injection with vial and syringe	Drug: Placebo; Subcutaneous injection of recommended dose of placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo	At week 52

Secondary Outcome Measures

Outcome Measure	Time Frame
Change from baseline in standing height at Week 52 versus placebo	At week 52
Change from baseline in height Z-score at Week 52 versus placebo	At week 52

Collaborators and Investigators

• Sponsor: BioMarin Pharmaceutical
• Investigators: Study Director: Medical Director, MD, BioMarin Pharmaceutical

Study record dates

Study Major Dates

• Study Start (Actual): June 17, 2024
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): August 1, 2026

Study Registration Dates

• First Submitted: June 7, 2024
• First Submitted That Met QC Criteria: June 7, 2024
• First Posted (Actual): June 12, 2024

Study Record Updates

• Last Update Posted (Actual): January 20, 2026
• Last Update Submitted That Met QC Criteria: January 15, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: Musculoskeletal Diseases; Osteochondrodysplasias; Genetic Diseases, Inborn; Physiological Effects of Drugs; Bone Diseases; Dwarfism; Skeletal Dysplasias; Natriuretic Agents; Natriuretic Peptide, C-Type; Hypochondroplasia; Vosoritide; Voxzogo; HCH; Bone Disease, Developmental
• Additional Relevant MeSH Terms: Endocrine System Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Musculoskeletal Diseases; Bone Diseases; Dwarfism; Osteochondrodysplasias; Bone Diseases, Developmental; Hypochondroplasia; Physiological Effects of Drugs; Natriuretic Agents; vosoritide
• Other Study ID Numbers: 111-303

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No