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An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGPHASE2

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

A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia

NCT03989947•BioMarin Pharmaceutical

View on ClinicalTrials.gov

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

Age

1 - No limit years

Sex

ALL

Healthy Volunteers

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 compliance

Locations (16)

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

Key Dates

Start Date

June 12, 2019

Primary Completion Date

May 1, 2038

Completion Date

May 1, 2038

Last Updated

March 13, 2026

Enrollment

ACTUAL participants

Conditions

Achondroplasia

Interventions

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

DRUG

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

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Achondroplasia

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ENROLLING_BY_INVITATIONPHASE2/PHASE3

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TERMINATEDPHASE2

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 13, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia

A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia

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Radiological Analysis on Patients With Achondroplasia Disorder