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

Achondroplasia Clinical Trial

— 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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02724228
• Other study ID #: 111-205
• Secondary ID: 2015-004004-30
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: January 26, 2016
• Est. completion date: February 2028

Study information

• Verified date: December 2023
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 30
• Est. completion date: February 2028
• Est. primary completion date: December 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 7 Years and older

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.

Related Conditions & MeSH terms

• Achondroplasia

Intervention

Drug:
• BMN 111
BMN 111 will be administered subcutaneously daily.

Locations

Country	Name	City	State
Australia	Murdoch Children's Research Institute	Parkville	Victoria
France	Institut Necker	Paris
United Kingdom	Guys & St. Thomas NHS Foundation Trust Evelina Hospital	London
United States	Johns Hopkins McKusick - Institute of Genetic Medicine	Baltimore	Maryland
United States	Ann and Robert H. Lurie Childrens Hospital of Chicago	Chicago	Illinois
United States	Baylor College of Medicine	Houston	Texas
United States	Vanderbilt University	Nashville	Tennessee
United States	Children's Hospital & Research Center Oakland	Oakland	California
United States	Harbor - UCLA Medical Center	Torrance	California

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

United States,  Australia,  France,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Growth Velocity [Efficacy]	Annualized growth velocity (cm/yr)	Until near final adult height is reached
Secondary	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
Secondary	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
Secondary	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