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New hope for kids with dwarfism: daily shot may boost growth

NCT ID NCT03989947

Disease control Sponsor: BioMarin Pharmaceutical Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a daily injection called BMN 111 in about 70 children with achondroplasia, the most common form of dwarfism. The goal is to see if the drug is safe and helps children grow taller over several years. All participants had already received BMN 111 or a placebo in a previous study and will now receive the active drug.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

73 people

The number who actually took part.

Started

Jun 2019

Expected to finish

May 2038

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

15 months and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

achondroplasia bone disorder Dwarfism musculoskeletal system disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Achondroplasia

Contacts and locations

Locations

  • Alfred I. duPont Hospital for Children

    Wilmington, Delaware, 19803, United States

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

    Chicago, Illinois, 60611, United States

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Children's Hospital & Research Center Oakland

    Oakland, California, 94609, United States

  • Cincinnati Childrens Hospital

    Cincinnati, Ohio, 45229, United States

  • Emory University

    Decatur, Georgia, 30033, United States

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

    London, SE19RT, United Kingdom

  • Harbor - UCLA Medical Center

    Torrance, California, 90509, United States

  • Medical College of Wisconsin, Children's Hospital

    Milwaukee, Wisconsin, 53226, United States

  • Murdoch Children's Research Institute

    Parkville, Victoria, 3052, Australia

  • Osaka University

    Osaka, 565-0871, Japan

  • Saitama Children's Medical Center

    Saitama, 330-8777, Japan

  • Sheffield Children's NHS Foundation Trust

    Sheffield, S102TH, United Kingdom

  • The Children's Hospital at Westmead

    Westmead, New South Wales, 2145, Australia

  • Tokushima University Hospital

    Tokushima, 77-8503, Japan

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232-2578, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.