BMN-111 Phase 2: study 111-202 and extension study 111-205

Study 111-202

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children with Achondroplasia (ACH)

This study is enrolling participants by invitation only

Estimated Enrollment: 36

Study start date: January 2014

Estimated Study Completion Date: December 2017

Estimated Primary Completion Date: November 2017

Arms	Assigned Interventions
Experimental: BMN 111 - Subcutaneous Injection 111-202 is an open-label sequential cohort dose-escalation study. BMN 111 will be administered as a morning dose in one of the following daily dosing regimens: Cohort 1: 2.5 microgram/kg, Cohort 2: 7.5 microgram/kg, Cohort 3: 15.0 microgram/kg, Cohort 4: 30.0 microgram/kg Cohort 5: up to 60.0 microgram/kg	Drug: BMN 111   BMN 111 will be administered daily for 24 months in an open-label sequential dose adjustment fashion.

Ages Eligible for Study:	5 Years to 14 Years
Genders Eligible for Study:	All
Accepts Healthy Volunteers:	No

Study 111-205

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children with Achondroplasia (ACH)

This study is enrolling participants by invitation only - is an extension study

Estimated Enrollment: 30

Study Start Date: January 2016

Estimated Study Completion Date: December 2022

Estimated Primary Completion Date: October 2022

Arms	Assigned Interventions
Experimental: BMN 111 - Subcutaneous Injection 111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study. BMN 111 will be administered in one of the following  daily dosing regimens: 15  µg/kg 30  µg/kg up to 60  µg/kg daily	Drug: BMN 111   BMN 111 will be administered daily for up to 260 weeks - 5 years

Ages Eligible for Study:	7 Years to 16 Years
Genders Eligible for Study:	All
Accepts Healthy Volunteers:	No