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

Phase 2

Active, not recruiting

• Conditions: Achondroplasia
• Interventions: Drug: BMN 111

• Registration Number: NCT02724228
• Lead Sponsor: BioMarin Pharmaceutical

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-01-26
• Study First Submitted: 2016-02-12
• Study First Submitted QC: 2016-03-25
• Study First Posted: 2016-03-31
• Status Verified: 2024-12
• Last Update Submitted: 2025-01-07
• Last Update Posted: 2025-01-09
• Primary Completion: 2027-12
• Study Completion: 2028-02

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 30

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
BMN 111 - Subcutaneous Injection	BMN 111	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.

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]	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 Outcome Measures

Name	Time	Method
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	Upper-to-lower body segment ratio
Growth Velocity [Efficacy]	Until near final adult height is reached	Annualized growth velocity (cm/yr)
Final Adult Height	Up to at least 16 years of age for females and 18 years of age for males	Height at the age of 16 years for females and 18 years 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	Height standard score (Z-score)

Trial Locations

Locations (9)

Ann and Robert H. Lurie Childrens Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

Children's Hospital & Research Center Oakland; 🇺🇸 Oakland, California, United States

Harbor - UCLA Medical Center; 🇺🇸 Torrance, California, United States

Guys & St. Thomas NHS Foundation Trust Evelina Hospital; 🇬🇧 London, United Kingdom

Institut Necker; 🇫🇷 Paris, France

Johns Hopkins McKusick - Institute of Genetic Medicine; 🇺🇸 Baltimore, Maryland, United States

Baylor College of Medicine; 🇺🇸 Houston, Texas, United States

Vanderbilt University; 🇺🇸 Nashville, Tennessee, United States

Murdoch Children's Research Institute; 🇦🇺 Parkville, Victoria, Australia