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

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Trial Status Completed

Trial Identifier

NCT03197766

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

5 Years to 18 Years

Healthy Volunteers

No

Drug/Treatment:

BMN 111, Placebo

Phase:

Phase 3

Study Type:

Interventional

Number of Participants:

121

Study Started:

2016-12-12

Study Updated:

2022-03-02

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

    Decatur, 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-Guericke 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

Eligibility Criteria

Inclusion Criteria

* Parent(s) or guardian(s) consent
* 5 to 2 mg/dL
* Chronic anemia
* Baseline systolic blood pressure (BP) 450 msec
* Have an unstable condition likely to require surgical intervention during the study (including progressive cervical medullary compression or severe untreated sleep apnea)
* Decreased growth velocity (< 1.5 cm/yr) over a period of 6 months or evidence of growth plate closure (proximal tibia, distal femur) * Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or treatment greater than 6 months at any time * Greater than 1 month treatment with oral corticosteroids (low-dose ongoing inhaled steroid for asthma, or intranasal steroids, are acceptable) in the previous 12 months * Planned or expected to have limb-lengthening surgery during the study period. Subjects with previous limb- lengthening surgery may enroll if surgery occurred at least 18 months prior to the study and healing is complete without sequelae. * Planned or expected bone-related surgery (ie. surgery involving disruption of bone cortex, excluding tooth extraction), during the study period. Subjects with previous bone-related surgery may enroll if surgery occurred at least 6 months prior to the study and healing is complete without sequelae. * Had a fracture of the long bones or spine within 6 months prior to screening * History of severe untreated sleep apnea * New initiation of sleep apnea treatment (e.g. CPAP or sleep apnea-mitigating surgery) in the previous 2 months prior to screening * History of hip surgery or hip dysplasia atypical for achondroplastic subjects * History of clinically significant hip injury in the 30 days prior to screening * History of slipped capital femoral epiphysis or avascular necrosis of the femoral head * Abnormal findings on baseline clinical hip exam or imaging assessments that are determined to be clinically significant * Concurrent disease or condition that would interfere with study participation or safety evaluations, for any reason * Condition or circumstance that places the subject at high risk for poor treatment compliance or for not completing the study

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