A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

Status:	Enrolling by invitation
Healthy:	No
Age Range:	Any
Updated:	2/17/2019
Start Date:	May 23, 2018
End Date:	May 2021

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia, Age 0 to < 60 Months

Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN
111 in infants and young children with a diagnosis of Achondroplasia.

Inclusion Criteria:

- Diagnosis of ACH, confirmed by genetic testing

- Age 0 to < 60 months at study entry (Day 1)

- At least 6-month period of pretreatment growth assessment in Study 111-901 immediately
before study entry (cohort 1 & 2) or at least 3 months of observation prior to
treatment (cohort 3)

Exclusion Criteria:

- Subject weight < 5.0 kg (cohort 1 & 2) or < 4.0 kg (cohort 3)

- Treatment with growth hormone within 6-months prior to screening or prolonged
treatment (> 3 months) at any time

- Any history of spine or long-bone surgery or any bone-related surgery with chronic
complications

- Any history of limb-lengthening surgery or planned limb-lengthening during the study

- Fracture of the long bones within 6 months prior to screening

We found this trial at

sites

Children's Hospital at Westmead

Westmead, New South Wales 2145

from

Westmead,