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

BioMarin Pharmaceutical9 sites in 4 countries30 target enrollmentJanuary 26, 2016

ConditionsAchondroplasia

InterventionsBMN 111

DrugsBMN 111

Overview

Phase: Phase 2
Intervention: BMN 111
Conditions: Achondroplasia
Sponsor: BioMarin Pharmaceutical
Enrollment: 30
Locations: 9
Primary Endpoint: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Status: Active, Not Recruiting
Last Updated: last month

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

January 26, 2016

End Date

February 1, 2028

Last Updated

last month

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

BioMarin Pharmaceutical

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Have completed 24 months of BMN 111 treatment in Study 111-
•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.

Arms & Interventions

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, initially up to 30 μg/kg. BMN 111 will be administered by weight-band dosing regimen.

Intervention: BMN 111

Outcomes

Primary Outcomes

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

Time Frame: 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

* Number of study participants with treatment-emergent adverse events. * Number of study participants with treatment-emergent serious adverse events

Secondary Outcomes

Body Proportions (Efficacy)(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)
Growth Velocity [Efficacy](Until near final adult height is reached)
Final Adult Height(Up to at least 16 years of age for females and 18 years of age for males)
Growth Parameters (Efficacy)(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)