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

March 12, 2026 updated by: BioMarin Pharmaceutical

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
73

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Westmead, New South Wales, Australia, 2145
        • The Children's Hospital at Westmead
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • Murdoch Children's Research Institute
  • Japan
      • Osaka, Japan, 565-0871
        • Osaka University
      • Saitama, Japan, 330-8777
        • Saitama Children's Medical Center
      • Tokushima, Japan, 77-8503
        • Tokushima University Hospital
  • United Kingdom
      • London, United Kingdom, SE19RT
        • Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital
      • Sheffield, United Kingdom, S102TH
        • Sheffield Children's NHS Foundation Trust
  • United States
    • California
      • Oakland, California, United States, 94609
        • Children's Hospital & Research Center Oakland
      • Torrance, California, United States, 90509
        • Harbor - UCLA Medical Center
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Alfred I. DuPont Hospital for Children
    • Georgia
      • Decatur, Georgia, United States, 30033
        • Emory University
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann Robert and H. Lurie Children's Hospital of Chicago
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Childrens Hospital
    • Tennessee
      • Nashville, Tennessee, United States, 37232-2578
        • Vanderbilt University Medical Center
    • Texas
      • Houston, Texas, United States, 77030
        • Baylor College of Medicine
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin, Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Active BMN 111
Once daily subcutaneous injections of recommended dose of BMN 111 based on weight-band dosing.
Drug: Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Modified recombinant human C-type natriuretic peptide (subject to adjustment per protocol)
Other Names:
  • Vosoritide

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time Frame: "Through study completion, an average of 5 years"
Number of study participants with treatment-emergent adverse events or serious adverse events
"Through study completion, an average of 5 years"
Evaluate change in height/length z-score in children with ACH treated with BMN 111
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Evaluate the change from baseline of mean annualized growth velocity (AGV)
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"
Characterize maximum concentration (Cmax) of BMN 111 in plasma
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"
Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞)
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"
Characterize the elimination half-life of BMN 111 (t½)
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"
Characterize the apparent clearance of drug
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"
Characterize the apparent volume of distribution based upon the terminal phase (Vz/F)
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"
Characterize the amount of time BMN 111 is present at maximum concentration (Tmax)
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"
Evaluate the change from baseline on body proportion ratios of the extremities
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"
Effect of BMN 111 on bone morphology and quality by XRay
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"
The effect of BMN 111 on bone morphology/quality will be assessed by measuring bone mineral density via Dual X-ray Absorptiometry
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"
Potential Changes in health-related quality of life as measured by the quality of life in Short- statured youth
Time Frame: "Through study completion, an average of 5 years"
Evaluate the long-term effect of BMN 111 on health-related quality of life, developmental status and functional independence, using age-specific QoL and functional independence questionnaires (Bayley-III, WeeFIM, ITQOL, QoLISSY, PedsQL, Child Behavior Checklist 1.5-5 [CBCL 1.5-5], Child Behavior Checklist 6-18 [CBCL 6-18]).
"Through study completion, an average of 5 years"
BMN 111 activity will be assessed by measuring bone and collagen metabolism
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"
Describe the incidence of surgical and medical interventions related to achondroplasia
Time Frame: "Through study completion, an average of 5 years"
"Through study completion, an average of 5 years"
Assess effect on sleep disordered breathing by polysomnography in patients up to 5 years old.
Time Frame: "Through study completion, an average of 1 year"
"Through study completion, an average of 1 year"
Evaluate the effect of BMN 111 on skull and brain morphology, including foramen magnum, ventricular and brain parenchymal dimensions by MRI in patients up to 3 years old.
Time Frame: "Through study completion, an average of 1 year"
"Through study completion, an average of 1 year"

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
BioMarin Pharmaceutical

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director MD, BioMarin Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 12, 2019

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
May 1, 2038

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 1, 2038

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 22, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 16, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 18, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 13, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 12, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 111-208

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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