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Active, not recruitingNCT03424018Updated Mar 13, 2026

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

A Phase 3 interventional study of BMN 111 in Achondroplasia, sponsored by BioMarin Pharmaceutical. Active, not recruiting at 24 sites in 7 countries. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-03-13.

Sponsored by BioMarin Pharmaceutical · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
119
Allocation
Not applicable
Ages
6 Years and older
Sex
All
01

Study summary

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

02

Conditions studied

  • Achondroplasia

Keywords

  • ACH
  • Achondroplasia
  • Bone Diseases
  • Bone Diseases, Developmental
  • Dwarfism
  • Genetic Diseases, Inborn
  • Musculoskeletal Diseases
  • Natriuretic Agents
  • Natriuretic Peptide, C-Type
  • Osteochondrodysplasias
  • Physiological Effects of Drugs
  • Skeletal Dysplasias
03

Who can participate

Ages eligible
6 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
119 participants (actual)

Study arms

  • Experimental
    BMN 111

    Drug: BMN 111

Interventions

  • DrugBMN 111

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

    Also known as: Vosoritide, Modified recombinant human C-type natriuretic peptide

05

What researchers measure

Primary outcomes

  1. Change from baselines in mean annualized growth velocity

    Long term efficacy as measured by change in annualized growth velocity

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

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

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

    Time frame: Through study completion, every 12 months

  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

    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

    Time frame: Through study completion, every 12 months for DXA or 2 years for X-ray

  13. Final Adult Height

    Height at 16 years for females and 18 years for males

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

Other outcomes

  1. Optional exploratory genomic biomarker analysis

    Exploratory genomic analysis of genes associated with CNP signaling

    Time frame: Once through study completion

06

Study locations

24 sites
  • Children's Hospital & Research Center Oakland
    Oakland, California 94609, United States
  • Harbor - UCLA Medical Center
    Torrance, California 90509, United States
  • Alfred I. duPont Hospital for Children
    Wilmington, Delaware 19803, United States
  • Emory University
    Atlanta, Georgia 30322, United States
  • Ann and Robert H. Lurie Children's Hospital of Chicago
    Chicago, Illinois 60611, United States
  • Johns Hopkins University
    Baltimore, Maryland 21287, United States
  • University of Missouri
    Columbia, Missouri 65201, United States
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, Ohio 45229, United States
  • Baylor College of Medicine
    Houston, Texas 77030, United States
  • Seattle Children's Hospital
    Seattle, Washington 98105, United States
  • Medical College of Wisconsin, Children's Hospital
    Milwaukee, Wisconsin 53226, United States
  • The Children's Hospital at Westmead
    Westmead, New South Wales 2145, Australia
  • Murdoch Children's Research Institute
    Parkville, Victoria 3052, Australia
  • Otto-von-Gericke Universitaet, Universitaetskinderklinik
    Magdeburg, 39120, Germany
  • Universitätsklinikum Münster
    Münster, 48149, Germany
  • Osaka University Hospital
    Osaka, Japan
  • Saitama Children's Medical Center
    Saitama, Japan
  • Tokushima University Hospital
    Tokushima, Japan
  • Institut Catala de Traumatologica I Medicina de l'Esport
    Barcelona, 08028, Spain
  • Hospital Sant Joan de Deu
    Barcelona, 08950, Spain
  • Hospital Universitario Virgen de la Victoria
    Málaga, 29010, Spain
  • Acibadem University School of Medicine
    Istanbul, 34752, Turkey (Türkiye)
  • Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital
    London, SE1 9RT, United Kingdom
  • Sheffield Children's NHS Foundation Trust
    Sheffield, S10 2TH, United Kingdom
07

References and documents

Publications

  • Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Palm K, Prada CE, Kubota T, Arundel P, Kotani Y, Leiva-Gea A, Bober MB, Hecht JT, Legare JM, Lawrinson S, Low A, Sabir I, Huntsman-Labed A, Day JRS. Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. Med. 2025 May 9;6(5):100566. doi: 10.1016/j.medj.2024.11.019. Epub 2024 Dec 30. PubMed 39740666 ↗
  • Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Mohnike K, Prada CE, Kubota T, Arundel P, Leiva-Gea A, Rowell R, Low A, Sabir I, Huntsman-Labed A, Day J. Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life. Genet Med. 2024 Dec;26(12):101274. doi: 10.1016/j.gim.2024.101274. Epub 2024 Sep 18. PubMed 39305160 ↗
  • Qi Y, Chan ML, Mould DR, Larimore K, Fisheleva E, Cherukuri A, Day J, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Bober MB, Henshaw J. Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model. Clin Pharmacokinet. 2024 May;63(5):707-719. doi: 10.1007/s40262-024-01371-6. Epub 2024 Apr 23. PubMed 38649657 ↗

Individual participant data

Plan to share: No

08

Registry details

Key details

Study ID
NCT03424018
Lead sponsor
BioMarin Pharmaceutical
Responsible party
Sponsor
First posted
Feb 6, 2018
Start date
Dec 12, 2017
Primary completion
Jun 2031 (estimated)
Completion
Jun 2031 (estimated)
Last update
Mar 13, 2026

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is active, not recruiting, as verified in Mar 2026. You cannot join it, but the record below documents what was studied.

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