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A phase 3, Open-Label, Long-Term extension study to evaluate the safety and efficacy of vosoritide in children with hypochondroplasia

NCT ID NCT07073014

NEW 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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 09, 2026 · Last updated Sep 09, 2026

Summary

The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH

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

About 140 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2025

Expected to finish

Oct 2044

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

Children (under 18), adults (18 to 64) and older adults (65 and over)

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: * Participants must have completed the Week 52 visit for 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays * Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any study-related procedure * Females ≥ 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, participants must be willing to use a highly effective method of contraception while participating in the study * Participants are willing and able to perform all study procedures as physically possible * Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training Exclusion Criteria: * Permanently discontinued study treatment in the studies 111-303 or 111-212 * Evidence of decreased growth velocity (AGV \< 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care * Taking or planning to take any prohibited medications * Planned or expected to have limb-lengthening surgery during the study period * Planned or expected bone-related surgery during the study period * Require any investigational agent prior to completion of study period * Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy * Have known hypersensitivity to vosoritide or its excipients * Is pregnant or breastfeeding at Baseline 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

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Conditions

The condition(s) this trial relates to.

hypochondroplasia

As listed by the trial registrant

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

Hypochondroplasia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    30 sites in 9 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

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

    Chicago, Illinois, 60611, United States

  • Benioff Children's Hospital - Oakland

    Oakland, California, 94609, United States

  • CHU de Toulouse

    Toulouse, France

  • Centre Hospitalier Universitaire Sainte-Justine

    Montreal, Quebec, Canada

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Cook Children's Endocrinology

    Fort Worth, Texas, 76104, United States

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Rome, Italy

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, United Kingdom

  • Hopital Necker-Enfants Malade

    Paris, France

  • Hopital de la Timone

    Marseille, France

  • Hospices Civils de Lyon - Hopital Femme Mere Enfant

    Lyon, France

  • Hospital MiKS - Unidad de Cirugía Artroscópica

    Vitoria-Gasteiz, Spain

  • Institute of Science Tokyo Hospital

    Tokyo, Japan

  • Instituto Giannina Gaslini

    Genoa, Italy

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Murdoch Children's Research Institute of The Royal Children's Hospital

    Parkville, Victoria, 3052, Australia

  • Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)

    Wilmington, Delaware, 19803, United States

  • Osaka University Hosptial

    Osaka, Japan

  • Ospedale Pediatrico Bambino Gesu

    Rome, Italy

  • Phoenix Children's Hospital - Thomas Campus

    Phoenix, Arizona, 85016, United States

  • SickKids - The Hospital for Sick Children

    Toronto, Ontario, Canada

  • The Johns Hopkins University School of Medicine

    Baltimore, Maryland, 21205, United States

  • Tokushima University Hospital

    Tokushima, Japan

  • Tottori University Hospital

    Tottori, Japan

  • Uniklinik Koln

    Cologne, Germany

  • Univeristatskinderklinik Magdeburg

    Magdeburg, Germany

  • University of Alberta Stollery Children's Hospital

    Edmonton, Alberta, Canada

  • University of Minnesota Masonic Children's Hospital

    Minneapolis, Minnesota, 55454, United States

  • University of Missouri

    Columbia, Missouri, 65201, United States

  • Universitätsklinikum des Saarlandes

    Homburg, Saarland, 66421, Germany

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