AzTrials
AzTrials

AchondroplasiaPhase 2NCT03989947

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

A Phase 2 study testing Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. in people with Achondroplasia, run by BioMarin Pharmaceutical at 16 sites in 4 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

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. Read more about this study

Who is it for?
Ages 15 months and older
Conditions the study is for
Achondroplasia
What is being tested?
Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
73 participants
Where
16 sitesin 4 countries
Study dates
12 Jun 2019to May 2038
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 15 months and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

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

Who cannot join

  • Permanently discontinued BMN 111 or placebo prior to completion of Study 111-206
  • Have a clinically significant finding or arrhythmia on ECG that indicates abnormal cardiac function or conduction or QTc-F > 450 msec
  • Require any investigational agent (except BMN 111) prior to completion of study period
  • 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
  • Pregnant or planning to become pregnant (self or partner) at any time during the 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

Trial locations and contacts

16 sites in 4 countries.

16 sites in 4 countries

United States9 sites

  • Ann Robert and H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • Cincinnati Childrens Hospital

    Cincinnati, Ohio, 45229

  • Emory University

    Decatur, Georgia, 30033

  • Baylor College of Medicine

    Houston, Texas, 77030

  • Medical College of Wisconsin, Children's Hospital

    Milwaukee, Wisconsin, 53226

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232-2578

  • Children's Hospital & Research Center Oakland

    Oakland, California, 94609

  • Harbor - UCLA Medical Center

    Torrance, California, 90509

  • Alfred I. duPont Hospital for Children

    Wilmington, Delaware, 19803

Other trials in United States

Japan3 sites

  • Osaka University

    Osaka, 565-0871

  • Saitama Children's Medical Center

    Saitama, 330-8777

  • Tokushima University Hospital

    Tokushima, 77-8503

Other trials in Japan

Australia2 sites

  • Murdoch Children's Research Institute

    Parkville, Victoria, 3052

  • The Children's Hospital at Westmead

    Westmead, New South Wales, 2145

Other trials in Australia

United Kingdom2 sites

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

    London, SE19RT

  • Sheffield Children's NHS Foundation Trust

    Sheffield, S102TH

Other trials in United Kingdom

About this study

Official title: 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.

Conditions
Achondroplasia
Being tested
Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Keywords
Dwarfism, Bone Disease, ACH, Natriuretic Peptide C-Type, Musculoskeletal Diseases, Skeletal Dysplasias
Medical subject headings
Achondroplasia, Dwarfism, Bone Diseases, Musculoskeletal Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

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

    Also called Vosoritide

    Modified recombinant human C-type natriuretic peptide (subject to adjustment per protocol)

Study groups in NCT03989947
Study groupRoleWhat they receive
Active BMN 111ExperimentalOnce daily subcutaneous injections of recommended dose of BMN 111 based on weight-band dosing.
Receives: Drug: Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

    Number of study participants with treatment-emergent adverse events or serious adverse events

    Measured: "Through study completion, an average of 5 years"

  2. Evaluate change in height/length z-score in children with ACH treated with BMN 111

    Measured: "Through study completion, an average of 5 years"

Secondary outcomes (15)

  1. Evaluate the change from baseline of mean annualized growth velocity (AGV)

    Measured: "Through study completion, an average of 5 years"

  2. Characterize maximum concentration (Cmax) of BMN 111 in plasma

    Measured: "Through study completion, an average of 5 years"

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

    Measured: "Through study completion, an average of 5 years"

  4. Characterize the elimination half-life of BMN 111 (t½)

    Measured: "Through study completion, an average of 5 years"

  5. Characterize the apparent clearance of drug

    Measured: "Through study completion, an average of 5 years"

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

    Measured: "Through study completion, an average of 5 years"

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

    Measured: "Through study completion, an average of 5 years"

  8. Evaluate the change from baseline on body proportion ratios of the extremities

    Measured: "Through study completion, an average of 5 years"

  9. Effect of BMN 111 on bone morphology and quality by XRay

    Measured: "Through study completion, an average of 5 years"

  10. The effect of BMN 111 on bone morphology/quality will be assessed by measuring bone mineral density via Dual X-ray Absorptiometry

    Measured: "Through study completion, an average of 5 years"

  11. Potential Changes in health-related quality of life as measured by the quality of life in Short- statured youth

    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]).

    Measured: "Through study completion, an average of 5 years"

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

    Measured: "Through study completion, an average of 5 years"

  13. Describe the incidence of surgical and medical interventions related to achondroplasia

    Measured: "Through study completion, an average of 5 years"

  14. Assess effect on sleep disordered breathing by polysomnography in patients up to 5 years old.

    Measured: "Through study completion, an average of 1 year"

  15. 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.

    Measured: "Through study completion, an average of 1 year"

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Jun 2019
  2. Study start12 Jun 2019
  3. Primary completionMay 2038 (estimated)
  4. Study completionMay 2038 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
73 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03989947
Sponsor's protocol ID
111-208
Status last verified
Mar 2026
First submitted
22 May 2019
Last updated
13 Mar 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. in people with Achondroplasia, run by BioMarin Pharmaceutical at 16 sites in 4 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 13 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 16 sites in United States, Japan, Australia, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: "Through study completion, an average of 5 years"). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03989947.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Mar 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in