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

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

Conditions
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Achondroplasia

Intervention / Treatment
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* Drug: BMN 111


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Subjects under the age of majority are willing and able to provide written assent (if required). Subjects who reach the age of majority in their country will be asked to provide their own written consent upon reaching the legal age of majority. 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. Exclusion Criteria: 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 at Baseline visit or planning to become pregnant (self or partner) at any time during the study. Current chronic therapy with restricted medications.

Ages Eligible for Study
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Minimum Age: 7 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BMN 111 - Subcutaneous Injection<br>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, initially up to 30 μg/kg. BMN 111 will be administered by weight-band dosing regimen. | Drug: BMN 111<br>* BMN 111 will be administered subcutaneously daily.<br>* Other names: Vosoritide;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] | Number of study participants with treatment-emergent adverse events. Number of study participants with treatment-emergent serious adverse events | Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Growth Velocity [Efficacy] | Annualized growth velocity (cm/yr) | Until near final adult height is reached | 
| Growth Parameters (Efficacy) | Height standard score (Z-score) | Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later | 
| Body Proportions (Efficacy) | Upper-to-lower body segment ratio | Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later | 
| Final Adult Height | Height at the age of 16 years for females and 18 years for males | Up to at least 16 years of age for females and 18 years of age for males | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Achondroplasia, Dwarfism, Bone Diseases, Developmental, Bone Diseases