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Active, Not Recruiting

NCT Number: NCT03424018

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

The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Achondroplasia Bone Diseases Bone Diseases, Developmental Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dwarfism Endocrine System Diseases Genetic Diseases, Inborn Musculoskeletal Diseases Osteochondrodysplasias

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

The Children's Hospital at Westmead, Westmead, New South Wales, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

BMN 111

Drug

Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.

Other names: Vosoritide, Modified recombinant human C-type natriuretic peptide

Primary outcomes

  1. Change from baselines in mean annualized growth velocity

    Time frame: Through study completion, an average of 1 year

    Long term efficacy as measured by change in annualized growth velocity

Secondary outcomes

  1. 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

  2. Potential changes in daily activity performance as measured by Activities of Daily Living questionnaire

    Time frame: Through study completion, every 12 months

  3. Characterize maximum concentration (Cmax) of BMN 111 in plasma

    Time frame: Through study completion, every 12 months

  4. Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞)

    Time frame: Through study completion, every 12 months

  5. 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

  6. Characterize the elimination half-life of BMN 111 (t1⁄2)

    Time frame: Through study completion, every 12 months

  7. Characterize the apparent clearance of drug

    Time frame: Through study completion, every 12 months

  8. Characterize the apparent volume of distribution based upon the terminal phase (Vz/F)

    Time frame: Through study completion, every 12 months

  9. Characterize the amount of time BMN 111 is present at maximum concentration (Tmax)

    Time frame: Through study completion, every 12 months

  10. BMN 111 Activity Biomarkers

    Time frame: Through study completion, every 12 months

    BMN 111 activity will be assessed by measuring bone and collagen metabolism

  11. Evaluate change from baseline in body proportion ratios of the extremities

    Time frame: Through study completion, every 6 months

  12. 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

    The effect of BMN 111 on bone morphology/quality will be assessed by measuring bone mineral density via X-Ray and Dual X-ray Absorptiometry

  13. Final Adult Height

    Time frame: Up to at least 16 years of age for females and 18 years of age for males

    Height at 16 years for females and 18 years for males

Other outcomes

  1. Optional exploratory genomic biomarker analysis

    Time frame: Once through study completion

    Exploratory genomic analysis of genes associated with CNP signaling

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study title

A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia

Important dates

Study start
2017
Primary completion
2031
Study completion
2031
First posted
Feb 6, 2018
Registry last updated
Mar 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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