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New drug aims to boost height in kids with dwarfism

NCT ID NCT07441876

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 21, 2026 · Updated 4 times

Summary

This study tests a new medicine called BMN 333 against an existing drug (vosoritide) to see if it can safely improve growth in children with achondroplasia, the most common form of dwarfism. About 160 children aged 2 to 17 will take part. The goal is to measure how much they grow over a year.

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 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 160 people

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

Started

Apr 2026

Expected to finish

Sep 2029

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

2 to 17 years

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. Participants must be aged ≥ 2 to \< 11 years (Phase 2) or ≥ 2 to \< 18 years (Phase 3), at the time of signing the informed consent 2. Participants must have ACH (confirmed by documented genetic testing) and open epiphyses 3. Are Tanner Stage I (Phase 2) or any Tanner stage (Phase 3) 4. Are ambulatory and able to stand without assistance Exclusion Criteria: 1. Have any short stature condition other than ACH (eg, hypochondroplasia, trisomy 21, pseudoachondroplasia, GH deficiency) 2. Have any of the following disorders: Hypothyroidism or hyperthyroidism, unless treated with evidence of normalized thyroid-stimulating hormone (TSH) levels, diabetes mellitus, unless considered well-controlled, autoimmune inflammatory disease, inflammatory bowel disease, autonomic neuropathy, anemia defined as hemoglobin \< 10 g/dL, vitamin D deficiency, significant hip pathology. 3. Have history of any renal insufficiency or cardiac/ cardiovascular disease that places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension. 4. Have had bone fractures of the long bones or spine within 6 months prior to screening. 5. Have used vosoritide, any other approved product (except GH, as detailed below), investigational product, or investigational medical device for the treatment of ACH or short stature at any time 6. Have been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start

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Conditions

The condition(s) this trial relates to.

achondroplasia bone disorder hereditary disease musculoskeletal system disorder osteochondrodysplasia

As listed by the trial registrant

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

Achondroplasia

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

    25 sites in 9 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: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • Alder Hey Childrens NHS Foundation Trust

    RECRUITING

    Liverpool, United Kingdom

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

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Children's Hospital of Philadelphia

    NOT_YET_RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Consano Clinical Research, LLC

    RECRUITING

    San Antonio, Texas, 78231, United States

  • Craiova Emergency Clinical County

    NOT_YET_RECRUITING

    Craiova, 200642, Romania

  • Great Ormond Street Hospital

    NOT_YET_RECRUITING

    London, United Kingdom

  • Hokkaido University Hospital

    NOT_YET_RECRUITING

    Hokkaido, Japan

  • Institutul National de Endocrinologie C.I.Parhon

    NOT_YET_RECRUITING

    Bucharest, Romania

  • Irccs Ospedale Gaslini Di Genova

    NOT_YET_RECRUITING

    Genova, 16147, Italy

  • Johns Hopkins Medicine

    RECRUITING

    Baltimore, Maryland, 21205, United States

  • Murdoch Children's Research Institute

    RECRUITING

    Parkville, Victoria, 3052, Australia

  • National Center for Child Health and Development

    RECRUITING

    Tokyo, Japan

  • Nemours Children's Health

    NOT_YET_RECRUITING

    Wilmington, Delaware, 19803, United States

  • Osaka City General Hospital

    RECRUITING

    Osaka, 534-0021, Japan

  • Pusan National University Yangsan Hospital

    NOT_YET_RECRUITING

    Yangsan, 50612, South Korea

  • Queen Elizabeth University Hospital

    RECRUITING

    Glasgow, United Kingdom

  • Seoul National University Hospital

    RECRUITING

    Seoul, South Korea

  • Texas Children Hospital, Baylor College of Medicine

    RECRUITING

    Houston, Texas, 77030, United States

  • UCSF Benioff Children's Hospital Oakland

    RECRUITING

    Oakland, California, 94609, United States

  • Universite de Montreal - Centre Hospitalier Universitaire Sainte-Justine

    NOT_YET_RECRUITING

    Montreal, Quebec, H3T 1C5, Canada

  • University Hospitals Bristol NHS Foundation Trust - Bristol Royal Hospital for Children

    RECRUITING

    Bristol, United Kingdom

  • University NHS Foundation Trust NIHR Wellcome Trust Clinical Research Facility

    RECRUITING

    Manchester, United Kingdom

  • University of Texas Southwestern Medical Center

    NOT_YET_RECRUITING

    Dallas, Texas, 75207, United States

  • Uniwersytecki Szpital Kliniczny im. J. Mikulicza-Radeckiego we Wroclawiu Klinika Pediatrii i Chorob Infekcyjnych

    NOT_YET_RECRUITING

    Wroclaw, Poland

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