A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide in Children with Hypochondroplasia

2024-517238-16-00 · tracked via the Priya Life Science EU tracker

Sponsor

Biomarin Pharmaceutical Inc.

Sponsor type

Pharmaceutical company

Therapeutic area

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Decision date

03/11/2025

Enrollment target

Sites

France, Spain, Italy, Germany

Condition studied

Hypochondroplasia

Investigational medicinal product(s)

Voxzogo 1.2 mg powder and solvent for solution for injectionVoxzogo 0.4 mg powder and solvent for solution for injectionVoxzogo 0.56 mg powder and solvent for solution for injection

Eligibility

Age group

0-17 years

Sex

Female, Male

Primary endpoint

1. Incidence of treatment emergent adverse events over time until FAH, 2. Change from baseline in height Z-score yearly until FAH, 3. Change from baseline in height yearly until FAH

Endpoint detail

1. Change from baseline in height at Week 52 of Study 111-308, 2. Change from baseline in height Z-score at Week 52 of Study 111-308 Baseline is based on first dose of study drug (vosoritide or placebo) in the parent study (111-303 or 111 212).

Official registry record

This page summarises publicly available CTIS data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View 2024-517238-16-00 on CTIS ↗ ← All trials in the EU