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

Last updated: March 12, 2026
Sponsor: BioMarin Pharmaceutical
Overall Status: Active - Not Recruiting

Phase

3

Condition

Bone Diseases

Musculoskeletal Diseases

Severe Short Stature

Treatment

BMN 111

Clinical Study ID

NCT03424018
111-302
2017-002404-28
  • Ages > 6
  • All Genders

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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Must have completed Study 111-301

  • Female >= 10 years old or who have begun menses must have a negative pregnancy testat the Baseline Visit and be willing to have additional pregnancy tests during thestudy

  • If sexually active, willing to use a highly effective method of contraception whileparticipating 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 informedconsent after the nature of the study has been explained and prior to performance ofany research-related procedure. Also, subjects under the age of majority are willingand 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 anyresearch-related procedure. Subjects who reach the age of majority in their countrywhile the study is ongoing will be asked to provide their own written consent againupon reaching the legal age of majority.

Exclusion

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 indicatesabnormal cardiac function

  • Evidence of decreased growth velocity (<1.5 cm/year) as assessed over a period of atleast 6 months or of growth plate closure (proximal tibia, distal femur) throughbilateral 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, wouldinterfere with study participation or safety evaluations, for any reason.

  • Have a condition or circumstance that, in the view of the investigator, places thesubject at high risk for poor treatment compliance or for not completing the study.

Study Design

Total Participants: 119
Treatment Group(s): 1
Primary Treatment: BMN 111
Phase: 3
Study Start date:
December 12, 2017
Estimated Completion Date:
June 30, 2031

Connect with a study center

  • The Children's Hospital at Westmead

    Westmead, New South Wales 2145
    Australia

    Site Not Available

  • Murdoch Children's Research Institute

    Parkville, Victoria 3052
    Australia

    Site Not Available

  • Otto-von-Gericke Universitaet, Universitaetskinderklinik

    Magdeburg, 39120
    Germany

    Site Not Available

  • Universitätsklinikum Münster

    Münster, 48149
    Germany

    Site Not Available

  • Osaka University Hospital

    Osaka,
    Japan

    Site Not Available

  • Saitama Children's Medical Center

    Saitama,
    Japan

    Site Not Available

  • Tokushima University Hospital

    Tokushima,
    Japan

    Site Not Available

  • Hospital Sant Joan de Deu

    Barcelona, 08950
    Spain

    Site Not Available

  • Institut Catala de Traumatologica I Medicina de l'Esport

    Barcelona, 08028
    Spain

    Site Not Available

  • Hospital Universitario Virgen de la Victoria

    Málaga, 29010
    Spain

    Site Not Available

  • Acibadem University School of Medicine

    Istanbul, 34752
    Turkey

    Site Not Available

  • Acibadem University School of Medicine

    Istanbul, 34752
    Turkey (Türkiye)

    Site Not Available

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

    London, SE1 9RT
    United Kingdom

    Site Not Available

  • Sheffield Children's NHS Foundation Trust

    Sheffield, S10 2TH
    United Kingdom

    Site Not Available

  • Children's Hospital & Research Center Oakland

    Oakland, California 94609
    United States

    Site Not Available

  • Harbor - UCLA Medical Center

    Torrance, California 90509
    United States

    Site Not Available

  • Alfred I. duPont Hospital for Children

    Wilmington, Delaware 19803
    United States

    Site Not Available

  • Emory University

    Atlanta, Georgia 30322
    United States

    Site Not Available

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

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Johns Hopkins University

    Baltimore, Maryland 21287
    United States

    Site Not Available

  • University of Missouri

    Columbia, Missouri 65201
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Site Not Available

  • Baylor College of Medicine

    Houston, Texas 77030
    United States

    Site Not Available

  • Seattle Children's Hospital

    Seattle, Washington 98105
    United States

    Site Not Available

  • Medical College of Wisconsin, Children's Hospital

    Milwaukee, Wisconsin 53226
    United States

    Site Not Available

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