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Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

NCT06455059 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 80

Last updated 2026-01-20

No results posted yet for this study

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).

Conditions

Interventions

DRUG

Vosoritide

Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.

DRUG

Placebo

Subcutaneous injection of recommended dose of placebo

Sponsors & Collaborators

Principal Investigators

  • Medical Director, MD · BioMarin Pharmaceutical

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-06-17
Primary Completion
2026-08-01
Completion
2026-08-01
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • France
  • Germany
  • Italy
  • Japan
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06455059 on ClinicalTrials.gov