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Hope for growth: new drug shows promise in Long-Term achondroplasia study

NCT ID NCT02724228

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 looks at the long-term safety and effects of a daily injection called BMN 111 in children with achondroplasia, a common form of dwarfism. It includes 30 children who have already taken the drug for two years in a previous study. The goal is to see if the drug remains safe and can help improve growth over time.

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

30 people

The number who actually took part.

Started

Jan 2016

Expected to finish

Feb 2028

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

7 years 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: * Have completed 24 months of BMN 111 treatment in Study 111-202. * Parent(s) or guardian(s) are willing and able to provide written, signed informed consent. Subjects under the age of majority are willing and able to provide written assent (if required). Subjects who reach the age of majority in their country will be asked to provide their own written consent upon reaching the legal age of majority. * If sexually active, willing to use a highly effective method of contraception while participating in the study. * Females \>= 10 years old or who have started menses must have a negative pregnancy test at baseline and be willing to have additional pregnancy tests during the study * Willing and able to perform all study procedures as physically possible * Parents/caregivers willing to administer daily injections to the subjects and complete the required training. Exclusion Criteria: * Requires any investigational agent prior to completion of study period. * 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. * Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation or safety evaluations for any reason. * Permanently discontinued BMN 111 during the 111-202 study. * Subject is pregnant at Baseline visit or planning to become pregnant (self or partner) at any time during the study. * Current chronic therapy with restricted medications.

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Conditions

The condition(s) this trial relates to.

achondroplasia Bone Diseases, Developmental bone disorder Dwarfism

As listed by the trial registrant

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

Achondroplasia

Contacts and locations

Locations

  • Ann and Robert H. Lurie Childrens 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

  • Guys & St. Thomas NHS Foundation Trust Evelina Hospital

    London, SE1 9RT, United Kingdom

  • Harbor - UCLA Medical Center

    Torrance, California, 90509, United States

  • Institut Necker

    Paris, 75015, France

  • Johns Hopkins McKusick - Institute of Genetic Medicine

    Baltimore, Maryland, 21287, United States

  • Murdoch Children's Research Institute

    Parkville, Victoria, 3052, Australia

  • Vanderbilt University

    Nashville, Tennessee, 37232-2578, United States

More trials for these conditions

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