Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New drug aims to help short-statured children grow taller

NCT ID NCT06455059

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 vosoritide to see if it can safely help children with hypochondroplasia grow faster. About 80 children aged 3 to 17 with a confirmed genetic diagnosis and very short stature will receive either the drug or a placebo for one year. The main goal is to measure how much their growth rate improves compared to those on placebo.

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 80 people

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

Started

Jun 2024

Expected to finish

Aug 2026

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

3 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 ≥ 3 to \< 18 years of age at enrollment 2. A confirmed genetic diagnosis of HCH 3. A height Z score of ≤ - 2.0 standard deviations (SDs) in reference to the general population of the same age and sex, as calculated using the Center for Disease Control and Prevention (CDC) growth charts 4. Males and females are eligible to participate in this clinical study. 5. Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study. 6. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study. Exclusion Criteria: 1. Short stature condition other than HCH 2. Have an unstable condition likely to require surgical intervention during the study. 3. Evidence of decreased growth velocity and/or growth plate closure 4. Taking any of the prohibited medications 5. Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids 6. Planned or expected to have limb-lengthening surgery during the study period. 7. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period 8. Require any investigational agent prior to completion of study period. 9. Received vosoritide or another investigational product or investigational medical device in the past 10. Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide 11. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy. 12. Have known hypersensitivity to vosoritide or its excipients. 13. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Hypochondroplasia are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

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

As listed by the trial registrant

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

Hypochondroplasia

Contacts and locations

Locations

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

    Chicago, Illinois, 60611, 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

  • 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

  • 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

    Parkville, Victoria, 3052, Australia

  • Nemours Alfred I. DuPont Hospital for Children

    Wilmington, Delaware, 19803, United States

  • Osaka University Hosptial

    Osaka, Japan

  • Ospedale Pediatrico Bambino Gesu

    Rome, Italy

  • SickKids - The Hospital for Sick Children

    Toronto, Ontario, Canada

  • 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

  • Vithas Hospital San Jose

    Vitoria-Gasteiz, Spain

More trials for these conditions

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