Clinical trial NCT02724228: A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
Official title: A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Achondroplasia
Keywords: Achondroplasia
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2016-03-31
Enrollment: 30
Sex: ALL
Minimum age (years): 7
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
timeFrame: 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 outcomes:
measure: Growth Velocity [Efficacy]
timeFrame: Until near final adult height is reached
measure: Growth Parameters (Efficacy)
timeFrame: 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
measure: Body Proportions (Efficacy)
timeFrame: 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
measure: Final Adult Height
timeFrame: Up to at least 16 years of age for females and 18 years of age for males
Other outcomes:
Locations:
city: Oakland
country: United States
facility: Children's Hospital & Research Center Oakland
state: California
city: Torrance
country: United States
facility: Harbor - UCLA Medical Center
state: California
city: Chicago
country: United States
facility: Ann and Robert H. Lurie Childrens Hospital of Chicago
state: Illinois
city: Baltimore
country: United States
facility: Johns Hopkins McKusick - Institute of Genetic Medicine
state: Maryland
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Parkville
country: Australia
facility: Murdoch Children's Research Institute
state: Victoria
city: Paris
country: France
facility: Institut Necker
city: London
country: United Kingdom
facility: Guys & St. Thomas NHS Foundation Trust Evelina Hospital
Study officials:
affiliation: BioMarin Pharmaceutical
name: Medical Director, MD
role: STUDY_DIRECTOR
References:
citation: Savarirayan R, Irving M, Bacino CA, Bostwick B, Charrow J, Cormier-Daire V, Le Quan Sang KH, Dickson P, Harmatz P, Phillips J, Owen N, Cherukuri A, Jayaram K, Jeha GS, Larimore K, Chan ML, Huntsman Labed A, Day J, Hoover-Fong J. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia. N Engl J Med. 2019 Jul 4;381(1):25-35. doi: 10.1056/NEJMoa1813446. Epub 2019 Jun 18.