Current partner codePEPTIDESDE
NCT03424018·Phase 3·INTERVENTIONAL

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

Status

Active, not recruiting

Phase

Phase 3

Enrollment

119

Locations

24

Results

Not posted

Publications

3

Study summary

What the protocol is testing.

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

Interventions

Treatment arms and agents.

DRUG

BMN 111

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

Timeline

From registration to results.

  1. First posted

    Feb 6, 2018

  2. Study start

    Dec 12, 2017

  3. Primary completion

    Jun 2031

  4. Study completion

    Jun 2031

  5. Results posted

    Not reported

  6. Registry updated

    Mar 13, 2026

Outcomes

What the study measures.

Primary outcomes

Change from baselines in mean annualized growth velocity

Time frame · Through study completion, an average of 1 year

Long term efficacy as measured by change in annualized growth velocity

Secondary outcomes

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

Time frame · Through study completion, every 6-12 months

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

Time frame · Through study completion, every 12 months

Characterize maximum concentration (Cmax) of BMN 111 in plasma

Time frame · Through study completion, every 12 months

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

Time frame · Through study completion, every 12 months

Characterize the area under the plasma concentration time-curve from time 0 to the last measurable concentration (AUC0-t)

Time frame · Through study completion, every 12 months

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

Time frame · Through study completion, every 12 months

Characterize the apparent clearance of drug

Time frame · Through study completion, every 12 months

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

Time frame · Through study completion, every 12 months

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

Time frame · Through study completion, every 12 months

BMN 111 Activity Biomarkers

Time frame · Through study completion, every 12 months

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

Eligibility

Who can take part.

Minimum age
6 Years
Maximum age
Not reported
Sex
ALL
Healthy volunteers
No

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.

Study locations

24 registered sites.

Australia · Germany · Japan · Spain · Turkey (Türkiye) · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.

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 (Türkiye)

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

London, United Kingdom

Sheffield Children's NHS Foundation Trust

Sheffield, United Kingdom

Related trials

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Related PeptideStat pages

Put the record in context.

Research pages describe evidence. Vendor pages, where available, describe independently tracked research-product listings and are not clinical recommendations.