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Growth drug shows promise for kids with dwarfism

NCT ID NCT03424018

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 drug called BMN 111 in children with achondroplasia, the most common form of dwarfism. The goal is to see if it safely helps them grow taller over several years. About 119 children who completed an earlier study will take the drug and be monitored for growth, daily activities, and quality of life.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

119 people

The number who actually took part.

Started

Dec 2017

Expected to finish

Jun 2031

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

6 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: * Must have completed Study 111-301 * Female \>= 10 years old or who have begun menses must have a negative pregnancy test at the Baseline Visit and be willing to have additional pregnancy tests during the study * If sexually active, willing to use a highly effective method of contraception while participating in the study * Are willing and able to perform all study procedures * 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. Exclusion Criteria: * Permanently discontinued BMN 111 or placebo prior to completion of the 111-301 study * Have a clinically significant finding or arrhythmia on Baseline ECG that indicates abnormal cardiac function * Evidence of decreased growth velocity (\<1.5 cm/year) as assessed over a period of at least 6 months or of growth plate closure (proximal tibia, distal femur) through bilateral lower extremity X-rays. * Require any investigational agent prior to completion of study period * Current therapy with medications known to alter renal function * Pregnant or breastfeeding or plan to become pregnant during 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 or for not completing the study.

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Conditions

The condition(s) this trial relates to.

achondroplasia Bone Diseases, Developmental bone disorder Dwarfism hereditary disease musculoskeletal system disorder osteochondrodysplasia

As listed by the trial registrant

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

Achondroplasia

Contacts and locations

Locations

  • Acibadem University School of Medicine

    Istanbul, 34752, Turkey (Türkiye)

  • Alfred I. duPont Hospital for Children

    Wilmington, Delaware, 19803, United States

  • Ann and Robert 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 Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Emory University

    Atlanta, Georgia, 30322, United States

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

    London, SE1 9RT, United Kingdom

  • Harbor - UCLA Medical Center

    Torrance, California, 90509, United States

  • Hospital Sant Joan de Deu

    Barcelona, 08950, Spain

  • Hospital Universitario Virgen de la Victoria

    Málaga, 29010, Spain

  • Institut Catala de Traumatologica I Medicina de l'Esport

    Barcelona, 08028, Spain

  • Johns Hopkins University

    Baltimore, Maryland, 21287, 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 Hospital

    Osaka, Japan

  • Otto-von-Gericke Universitaet, Universitaetskinderklinik

    Magdeburg, 39120, Germany

  • Saitama Children's Medical Center

    Saitama, Japan

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Sheffield Children's NHS Foundation Trust

    Sheffield, S10 2TH, United Kingdom

  • The Children's Hospital at Westmead

    Westmead, New South Wales, 2145, Australia

  • Tokushima University Hospital

    Tokushima, Japan

  • University of Missouri

    Columbia, Missouri, 65201, United States

  • Universitätsklinikum Münster

    Münster, 48149, Germany

More trials for these conditions

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