Summary

Eligibility
for people ages 15 months and up (full criteria)
Location
at UCLA UCSF
Dates
study started
study ends around

Description

Summary

This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height. Eligible subjects will have completed 1 year of BMN111 or placebo treatment in the 111-206 study and once enrolled in the 111-208 extension study will receive a daily dose of BMN111 by subcutaneous injection according to their age as determined by 111-206.

Official Title

A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia

Keywords

Achondroplasia, Dwarfism, Bone Disease, ACH, Natriuretic Peptide C-Type, Musculoskeletal Diseases, Skeletal Dysplasias, Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.

Eligibility

Locations

  • Children's Hospital & Research Center Oakland
    Oakland California 94609 United States
  • Harbor - UCLA Medical Center
    Torrance California 90509 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
BioMarin Pharmaceutical
ID
NCT03989947
Phase
Phase 2 Achondroplasia Research Study
Study Type
Interventional
Participants
About 73 people participating
Last Updated