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

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Trial Status Active Not Recruiting

Trial Identifier

NCT03989947

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

This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height. Eligible subjects will have completed 1 year of BMN111 or placebo treatment in the 111-206 study and once enrolled in the 111-208 extension study will receive a daily dose of BMN111 by subcutaneous injection according to their age as determined by 111-206.

Eligibility Criteria

Birth Sex

All

Age

From 15 Months

Healthy Volunteers

No

Drug/Treatment:

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

Phase:

Phase 2

Study Type:

Interventional

Number of Participants:

73

Study Started:

2019-06-12

Study Updated:

2025-01-09

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 Robert and H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, United States

  • Cincinnati Childrens Hospital

    Cincinnati, Ohio, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, United States

  • Baylor College of Medicine

    Houston, Texas, 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

  • Osaka University

    Osaka, , Japan

  • Saitama Children's Medical Center

    Saitama, , Japan

  • Tokushima University Hospital

    Tokushima, , Japan

  • 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

1. Must have completed Study 111-206 on investigational treatment (BMN 111 or placebo).
2. 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.
3. Are willing and able to perform all study procedures

Exclusion Criteria

1. Permanently discontinued BMN 111 or placebo prior to completion of Study 111-206
2. Have a clinically significant finding or arrhythmia on ECG that indicates abnormal cardiac function or conduction or QTc-F > 450 msec
3. Require any investigational agent (except BMN 111) prior to completion of study period
4. Current therapy with antihypertensive medications, angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium-channel blockers, cardiac glycosides, systemic anticholinergic agents, GnRH agonists, any medication that may impair or enhance compensatory tachycardia, diuretics, or other drugs known to alter renal or tubular function
5. Pregnant or planning to become pregnant (self or partner) at any time during the study
6. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason
7. Have a condition or circumstance that, in the view of the investigator, places the subject at high risk for poor treatment complianc

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