Interventional Study of Vosoritide for the Treatment of Children with Hypochondroplasia

Phase 3

• Conditions: Hypochondroplasia
• Interventions: Drug: Placebo; Drug: Vosoritide

• Registration Number: NCT06455059
• Lead Sponsor: BioMarin Pharmaceutical

Brief Summary

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).

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 Timeline

• Study Start: 2024-06-06
• Study First Submitted: 2024-06-07
• Study First Submitted QC: 2024-06-07
• Study First Posted: 2024-06-12
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-17
• Last Update Posted: 2025-03-19
• Primary Completion: 2026-08-01
• Study Completion: 2026-08-01

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 80

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Placebo injection with vial and syringe	Placebo	-
vosoritide injection with vial and syringe	Vosoritide	-

Primary Outcome Measures

Name	Time	Method
Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo	At week 52

Secondary Outcome Measures

Name	Time	Method
Change from baseline in height Z-score at Week 52 versus placebo	At week 52
Change from baseline in standing height at Week 52 versus placebo	At week 52

Trial Locations

Locations (23)

Nemours Alfred I. DuPont Hospital for Children; 🇺🇸 Wilmington, Delaware, United States

Ann & Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

Medical College of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States

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

Uniklinik Koln; 🇩🇪 Koln, 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

Vithas Hospital San Jose; 🇪🇸 Vitoria-Gasteiz, Spain

Great Ormond Street Hospital for Children NHS Foundation Trust; 🇬🇧 London, United Kingdom

Children's National Medical Center; 🇺🇸 Washington, District of Columbia, United States

Murdoch Children's Research Institute; 🇦🇺 Parkville, Victoria, Australia