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Summary

for people ages 7–16 (full criteria)
at Oakland, California and other locations
study started
estimated completion:

Description

Summary

This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.

Official Title

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

Keywords

Achondroplasia Dwarfism Bone Diseases, Developmental Bone Diseases Natriuretic Peptide, C-Type

Eligibility

You can join if…

Open to people ages 7–16

  • Have completed 24 months of BMN 111 treatment in Study 111-202.
  • Parent(s) or guardian(s) are willing and able to provide written, signed informed consent.
  • If sexually active, willing to use a highly effective method of contraception while participating in the study.
  • Females >= 10 years old or who have started menses must have a negative pregnancy test at baseline and be willing to have additional pregnancy tests during the study
  • Willing and able to perform all study procedures as physically possible
  • Parents/caregivers willing to administer daily injections to the subjects and complete the required training.

You CAN'T join if...

  • Requires any investigational agent prior to completion of study period.
  • Have a condition or circumstance that, in the view of the Investigator, places the subject at high risk for poor treatment compliance or for not completing the study.
  • Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation or safety evaluations for any reason.
  • Permanently discontinued BMN 111 during the 111-202 study.
  • Subject is pregnant.
  • Current chronic therapy with restricted medications.

Locations

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

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
BioMarin Pharmaceutical
ID
NCT02724228
Phase
Phase 2
Study Type
Interventional
Last Updated
June 8, 2017