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AchondroplasiaPhase 3NCT03424018

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

A Phase 3 study testing BMN 111 in people with Achondroplasia, run by BioMarin Pharmaceutical at 24 sites in 7 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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 Read more about this study

Who is it for?
Ages 6 years and older
Conditions the study is for
Achondroplasia
What is being tested?
BMN 111
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
119 participants
Where
24 sitesin 7 countries
Study dates
12 Dec 2017to Jun 2031
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

24 sites in 7 countries.

24 sites in 7 countries

United States11 sites

  • Emory University

    Atlanta, Georgia, 30322

  • Johns Hopkins University

    Baltimore, Maryland, 21287

  • Ann and Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • University of Missouri

    Columbia, Missouri, 65201

  • Baylor College of Medicine

    Houston, Texas, 77030

  • Medical College of Wisconsin, Children's Hospital

    Milwaukee, Wisconsin, 53226

  • Children's Hospital & Research Center Oakland

    Oakland, California, 94609

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • Harbor - UCLA Medical Center

    Torrance, California, 90509

  • Alfred I. duPont Hospital for Children

    Wilmington, Delaware, 19803

Other trials in United States

Japan3 sites

  • Osaka University Hospital

    Osaka

  • Saitama Children's Medical Center

    Saitama

  • Tokushima University Hospital

    Tokushima

Other trials in Japan

Spain3 sites

  • Institut Catala de Traumatologica I Medicina de l'Esport

    Barcelona, 08028

  • Hospital Sant Joan de Deu

    Barcelona, 08950

  • Hospital Universitario Virgen de la Victoria

    Málaga, 29010

Other trials in Spain

Australia2 sites

  • Murdoch Children's Research Institute

    Parkville, Victoria, 3052

  • The Children's Hospital at Westmead

    Westmead, New South Wales, 2145

Other trials in Australia

Germany2 sites

  • Otto-von-Gericke Universitaet, Universitaetskinderklinik

    Magdeburg, 39120

  • Universitätsklinikum Münster

    Münster, 48149

Other trials in Germany

United Kingdom2 sites

  • Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital

    London, SE1 9RT

  • Sheffield Children's NHS Foundation Trust

    Sheffield, S10 2TH

Other trials in United Kingdom

Turkey (Türkiye)1 site

  • Acibadem University School of Medicine

    Istanbul, 34752

Other trials in Turkey (Türkiye)

About this study

Official title: A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy 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

Conditions
Achondroplasia
Being tested
BMN 111
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
Medical subject headings
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, Dwarfism, Genetic Diseases, Inborn, Musculoskeletal Diseases, Osteochondrodysplasias

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BMN 111Drug

    Also called Vosoritide, Modified recombinant human C-type natriuretic peptide

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

Study groups in NCT03424018
Study groupRoleWhat they receive
BMN 111ExperimentalNot described
Receives: Drug: BMN 111

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change from baselines in mean annualized growth velocity

    Long term efficacy as measured by change in annualized growth velocity

    Measured: Through study completion, an average of 1 year

Secondary outcomes (13)

  1. Changes in health-related quality of life as measured by the Quality of Life in Short-Statured Youth questionnaire

    Measured: Through study completion, every 6-12 months

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

    Measured: Through study completion, every 12 months

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

    Measured: Through study completion, every 12 months

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

    Measured: 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)

    Measured: Through study completion, every 12 months

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

    Measured: Through study completion, every 12 months

  7. Characterize the apparent clearance of drug

    Measured: Through study completion, every 12 months

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

    Measured: Through study completion, every 12 months

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

    Measured: Through study completion, every 12 months

  10. BMN 111 Activity Biomarkers

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

    Measured: Through study completion, every 12 months

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

    Measured: 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

    Measured: 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

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Feb 2018
  2. Study start12 Dec 2017
  3. Primary completionJun 2031 (estimated)
  4. Study completionJun 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
119 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03424018
Sponsor's protocol ID
111-302
Other IDs
2017-002404-28, 2017
Status last verified
Mar 2026
First submitted
28 Dec 2017
Last updated
13 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

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

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing BMN 111 in people with Achondroplasia, run by BioMarin Pharmaceutical at 24 sites in 7 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 13 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 24 sites in United States, Japan, Spain, Australia, Germany and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from baselines in mean annualized growth velocity (time frame: Through study completion, an average of 1 year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03424018, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Mar 2026.

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