An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

• Register: ClinicalTrials.gov
• Last refreshed on: 27 January 2025
• Main ID: NCT03424018
• Date of registration: 28/12/2017
• Prospective Registration: No
• Primary sponsor: BioMarin Pharmaceutical
• Public title: An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
• Scientific title: A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia
• Date of first enrolment: December 12, 2017
• Target sample size: 119
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03424018
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Germany, Japan, Spain, Turkey, United Kingdom, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Must have completed Study 111-301

- Female >= 10 years old or who have begun menses must have a negative pregnancy test
at the Baseline Visit and be willing to have additional pregnancy tests during the
study

- If sexually active, willing to use a highly effective method of contraception while
participating in the study

- Are willing and able to perform all study procedures

- Parent(s) or guardian(s) are willing and able to provide written, signed informed
consent after the nature of the study has been explained and prior to performance of
any research-related procedure. Also, subjects under the age of majority are willing
and able to provide written assent (if required by local regulations or the IRB/IEC)
after the nature of the study has been explained and prior to performance of any
research-related procedure. Subjects who reach the age of majority in their country
while the study is ongoing will be asked to provide their own written consent again
upon reaching the legal age of majority.

Exclusion Criteria:

- Permanently discontinued BMN 111 or placebo prior to completion of the 111-301 study

- Have a clinically significant finding or arrhythmia on Baseline ECG that indicates
abnormal cardiac function

- Evidence of decreased growth velocity (<1.5 cm/year) as assessed over a period of at
least 6 months or of growth plate closure (proximal tibia, distal femur) through
bilateral lower extremity X-rays.

- Require any investigational agent prior to completion of study period

- Current therapy with medications known to alter renal function

- Pregnant or breastfeeding or plan to become pregnant during study

- Concurrent disease or condition that, in the view of the investigator, would
interfere with study participation or safety evaluations, for any reason.

- 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.


Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Achondroplasia

Intervention(s)

Drug: BMN 111

Primary Outcome(s)

Change from baselines in mean annualized growth velocity [Time Frame: Through study completion, an average of 1 year]

Secondary Outcome(s)

BMN 111 Activity Biomarkers [Time Frame: Through study completion, every 12 months]

Changes in health-related quality of life as measured by the Quality of Life in Short-Statured Youth questionnaire [Time Frame: Through study completion, every 6-12 months]

Characterize maximum concentration (Cmax) of BMN 111 in plasma [Time Frame: Through study completion, every 12 months]

Characterize the apparent volume of distribution based upon the terminal phase (Vz/F) [Time Frame: Through study completion, every 12 months]

Characterize the amount of time BMN 111 is present at maximum concentration (Tmax) [Time Frame: Through study completion, every 12 months]

Characterize the apparent clearance of drug [Time Frame: Through study completion, every 12 months]

Final Adult Height [Time Frame: Up to at least 16 years of age for females and 18 years of age for males]

Characterize the area under the plasma concentration time-curve from time 0 to the last measurable concentration (AUC0-t) [Time Frame: Through study completion, every 12 months]

Characterize the elimination half-life of BMN 111 (t1/2) [Time Frame: Through study completion, every 12 months]

Evaluate change from baseline in body proportion ratios of the extremities [Time Frame: Through study completion, every 6 months]

Potential changes in daily activity performance as measured by Activities of Daily Living questionnaire [Time Frame: Through study completion, every 12 months]

Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-8) [Time Frame: Through study completion, every 12 months]

Effect of BMN 111 on bone morphology and quality [Time Frame: Through study completion, every 12 months for DXA or 2 years for X-ray]

Secondary ID(s)

111-302

2017-002404-28

Source(s) of Monetary Support

Please refer to primary and secondary sponsors