BMN 111 for Achondroplasia

Recruiting in Palo Alto (17 mi)

+23 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: BioMarin Pharmaceutical

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia

Eligibility Criteria

This trial is for children with Achondroplasia who completed Study 111-301. They must be willing to use contraception if sexually active, and girls over 10 or menstruating need a negative pregnancy test. Participants should commit to study procedures, and minors require consent from guardians.

Inclusion Criteria

Must have completed Study 111-301

My guardian or I have agreed to participate in the study and signed the consent form.

If sexually active, willing to use a highly effective method of contraception while participating in the study

+2 more

Exclusion Criteria

I am taking medication that affects my kidney function.

I stopped taking BMN 111 or placebo before finishing the 111-301 study.

Require any investigational agent prior to completion of study period

+5 more

Participant Groups

The Phase 3 study tests BMN 111's effectiveness and safety in treating Achondroplasia in children. It aims to provide an additional therapeutic option by extending previous research findings from an earlier phase of the study.

1Treatment groups

Experimental Treatment

Group I: BMN 111Experimental Treatment1 Intervention