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

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Trial Status Active Not Recruiting

Trial Identifier

NCT03424018

Condition

Achondroplasia

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

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

Eligibility Criteria

Birth Sex

All

Age

From 6 Years

Healthy Volunteers

No

Drug/Treatment:

BMN 111

Phase:

Phase 3

Study Type:

Interventional

Number of Participants:

119

Study Started:

2017-12-12

Study Updated:

2025-03-25

Trial Locations

  • Children's Hospital & Research Center Oakland

    Oakland, California, United States

  • Harbor - UCLA Medical Center

    Torrance, California, United States

  • Alfred I. duPont Hospital for Children

    Wilmington, Delaware, United States

  • Emory University

    Atlanta, Georgia, United States

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

    Chicago, Illinois, United States

  • Johns Hopkins University

    Baltimore, Maryland, United States

  • University of Missouri

    Columbia, Missouri, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, United States

  • Baylor College of Medicine

    Houston, Texas, United States

  • Seattle Children's Hospital

    Seattle, Washington, United States

  • Medical College of Wisconsin, Children's Hospital

    Milwaukee, Wisconsin, United States

  • The Children's Hospital at Westmead

    Westmead, New South Wales, Australia

  • Murdoch Children's Research Institute

    Parkville, Victoria, Australia

  • Otto-von-Gericke Universitaet, Universitaetskinderklinik

    Magdeburg, , Germany

  • Universitätsklinikum Münster

    Münster, , 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, , Spain

  • Hospital Sant Joan de Deu

    Barcelona, , Spain

  • Hospital Universitario Virgen de la Victoria

    Málaga, , Spain

  • Acibadem University School of Medicine

    Istanbul, , Turkey

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

    London, , United Kingdom

  • Sheffield Children's NHS Foundation Trust

    Sheffield, , United Kingdom

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

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