DRUG
BMN 111
BMN 111 will be administered subcutaneously daily.
Status
Active, not recruiting
Phase
Phase 2
Enrollment
30
Locations
9
Results
Not posted
Publications
2
Study 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.
Interventions
DRUG
BMN 111 will be administered subcutaneously daily.
Timeline
First posted
Mar 31, 2016
Study start
Jan 26, 2016
Primary completion
Dec 2027
Study completion
Feb 2028
Results posted
Not reported
Registry updated
Mar 13, 2026
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
Growth Velocity [Efficacy]
Time frame · Until near final adult height is reached
Annualized growth velocity (cm/yr)
Growth Parameters (Efficacy)
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
Height standard score (Z-score)
Body Proportions (Efficacy)
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
Upper-to-lower body segment ratio
Final Adult Height
Time frame · 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
Eligibility
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 locations
Australia · France · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
Children's Hospital & Research Center Oakland
Oakland, California, United States
Harbor - UCLA Medical Center
Torrance, California, United States
Ann and Robert H. Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States
Johns Hopkins McKusick - Institute of Genetic Medicine
Baltimore, Maryland, United States
Vanderbilt University
Nashville, Tennessee, United States
Baylor College of Medicine
Houston, Texas, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
Institut Necker
Paris, France
Guys & St. Thomas NHS Foundation Trust Evelina Hospital
London, United Kingdom
Publications
Related trials
BioMarin · Achondroplasia
Phase 2 / Phase 3
Recruiting
160
2026-07
BioMarin · Idiopathic Short Stature
Phase 2
Recruiting
100
2026-05
BioMarin · Noonan Syndrome
Phase 2
Recruiting
30
2026-05
BioMarin · Hypochondroplasia
Phase 2
Recruiting
60
2026-03
Andrew Dauber · Short Stature
Phase 2
Active, not recruiting
56
2026-03
BioMarin · Achondroplasia
Phase 3
Active, not recruiting
119
2026-03
BioMarin · Achondroplasia
Phase 2
Active, not recruiting
73
2026-03
BioMarin · Achondroplasia
Phase 2
Active, not recruiting
20
2026-03
Related PeptideStat pages
Research pages describe evidence. Vendor pages, where available, describe independently tracked research-product listings and are not clinical recommendations.