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New hope for babies with rare dwarfism: daily shot may boost growth

NCT ID NCT07126262

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 study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 vosoritide in infants and toddlers (0 to 3 years old) with hypochondroplasia, a genetic condition that causes short stature. The goal is to see if the drug safely improves growth over 52 weeks. About 60 children will receive either vosoritide or a placebo, and their height, bone density, and side effects will be tracked.

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

About 60 people

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

Started

Jul 2025

Expected to finish

Jun 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

0 to 36 months

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.

Key Inclusion Criteria: 1. Participants must be 0 to \< 36 months of age at randomization. 2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH). 3. Participants aged 0 to \< 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to \< 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts. 4. Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg. Key Exclusion Criteria: 1. Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia). 2. Have an unstable medical condition likely to require surgical intervention during the study period. 3. Taking any of the prohibited medications. 4. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (\> 3 months) at any time. 5. Require any investigational agent prior to completion of study period. 6. Have received another investigational product or investigational medical device within 30 days prior to the Screening visit. 7. Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time. 8. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy. 9. Have known hypersensitivity to vosoritide or its excipients. 10. Have a condition or circumstance that, in the view of the investigator, places the participant at high risk for poor treatment compliance or for not completing the study. 11. Have any concurrent disease or condition that, in the view of the investigator, will 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 study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    26 sites in 7 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

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

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Benioff Children's Hospital - Oakland

    RECRUITING

    Oakland, California, 94609, United States

  • Cedars-Sinai Medical Center

    NOT_YET_RECRUITING

    Los Angeles, California, 90048, United States

  • Children's Health Queensland Hospital and Health Service

    NOT_YET_RECRUITING

    South Brisbane, Queensland, 4101, Australia

  • Children's National Medical Center

    RECRUITING

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

  • Children's Wisconsin - Fox Valley Hospital

    NOT_YET_RECRUITING

    Neenah, Wisconsin, 54956, United States

  • Cook Children's Endocrinology

    NOT_YET_RECRUITING

    Fort Worth, Texas, 76104, United States

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    NOT_YET_RECRUITING

    Roma, Rome, 00168, Italy

  • Great Ormond Street Hospital

    NOT_YET_RECRUITING

    London, England, WC1N3BH, United Kingdom

  • Hospices Civils de Lyon - Hôpital Femme Mère Enfant

    NOT_YET_RECRUITING

    Bron, Rhone-Alps, 69500, France

  • Hôpital Bicêtre

    RECRUITING

    Le Kremlin-Bicêtre, Paris, 94270, France

  • IRCCS Istituto Giannina Gaslini

    RECRUITING

    Genova, 16147, Italy

  • Institute of Science Tokyo Hospital

    RECRUITING

    Tokyo, 113-8519, Japan

  • Kumamoto University Hospital

    RECRUITING

    Kumamoto, 860-8556, Japan

  • Myriad Trials

    NOT_YET_RECRUITING

    London, England, W1W 5DT, United Kingdom

  • Nihon University Itabashi Hospital

    RECRUITING

    Tokyo, 173-8610, Japan

  • Osaka Women's and Children's Hospital

    RECRUITING

    Osaka, 94-1101, Japan

  • Phoenix Children's Hospital - Thomas Campus (Main)

    RECRUITING

    Phoenix, Arizona, 85016, United States

  • Royal Children's Hospital Melbourne

    RECRUITING

    Parkville, Victoria, 3052, Australia

  • The Johns Hopkins University School of Medicine

    RECRUITING

    Baltimore, Maryland, 21205, United States

  • Tottori University Hospital

    RECRUITING

    Tottori, 683-0854, Japan

  • Uniklinik Köln

    RECRUITING

    Cologne, North Rhine-Westphalia, 50937, Germany

  • University of Minneasota Masonic Children's Hospital

    RECRUITING

    Minneapolis, Minnesota, 55454, United States

  • University of Missouri

    RECRUITING

    Columbia, Missouri, 65211, United States

  • Universitätskinderklinik Magdeburg

    NOT_YET_RECRUITING

    Magdeburg, Saxony-Anhalt, 39120, Germany

  • Universitätsklinikum des Saarlandes

    RECRUITING

    Homburg, Saarland, 66421, Germany

More trials for these conditions

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