Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children With Hypochondroplasia

NCT06455059•BioMarin Pharmaceutical

View on ClinicalTrials.gov

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.

Eligibility

Age

3 - 17 years

Sex

ALL

Healthy Volunteers

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
+3 more criteria

Locations (23)

Nemours Alfred I. DuPont Hospital for Children

Wilmington, Delaware, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Murdoch Children's Research Institute

Parkville, Victoria, 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

Key Dates

Start Date

June 17, 2024

Primary Completion Date

August 1, 2026

Completion Date

August 1, 2026

Last Updated

January 20, 2026

Enrollment

ESTIMATED participants

Conditions

Hypochondroplasia

Interventions

Vosoritide

DRUG

Placebo

DRUG