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A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

NCT04554940 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2026-03-13

No results posted yet for this study

Summary

Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery

Conditions

Interventions

BIOLOGICAL

vosoritide

Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.

Sponsors & Collaborators

Principal Investigators

  • Medical Director, MD · BioMarin Pharmaceutical

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
0 Months
Max Age
12 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-10-10
Primary Completion
2027-12-31
Completion
2027-12-31
FDA Drug
Yes

Countries

  • Australia
  • United Kingdom

Study Locations

More Related Trials

Entities

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 NCT04554940 on ClinicalTrials.gov