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New drug aims to boost growth in kids with noonan syndrome

NCT ID NCT06668805

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Aug 25, 2026 · Updated 3 times

Summary

This study tests vosoritide, a protein that stimulates bone growth, in 30 children with Noonan syndrome who are not growing well despite growth hormone treatment. The goal is to see if vosoritide can safely increase their growth rate over six months. Participants will receive one of three doses, and researchers will monitor height changes and any side effects.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Vosoritide (a modified protein that helps with bone growth)
What this could lead to
If successful, vosoritide could offer a new treatment option to help children with Noonan syndrome grow taller when growth hormone alone isn't enough.
What could go wrong
This is an early Phase 2 trial with only 30 participants, so results may not apply to everyone. The long-term safety and effectiveness are still unknown, and there may be side effects.

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 30 people

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

Started

Nov 2024

Expected to finish

Sep 2041

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 11 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 years old, and \< 11 years old (females) or \< 12 years old (males), at the time of signing the informed consent form 2. A clinical diagnosis of Noonan syndrome. 3. A height assessment corresponding to a height Z-score of ≤ -1.28 SDs (below the 10th percentile for height) in reference to the general population of the same age and sex. 4. Tanner Stage 1, at time of signing the ICF. 5. Previous or current hGH treatment for short stature associated with their condition. 6. Inadequate growth confirmed with an AGV that is less than age- and sex-matched average stature AGV determined using median heights from CDC growth charts Exclusion Criteria: 1. Diagnosis of systemic disease or condition that may cause short stature other than Noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease. 2. Bone age advanced beyond chronological age by more than 2 years. 3. Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension, 4. Have an unstable condition likely to require surgical intervention during the study. 5. 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 using bilateral lower extremity X-rays. 6. Previous limb-lengthening surgery, or 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.

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Conditions

The condition(s) this trial relates to.

bone disorder Dwarfism musculoskeletal system disorder Noonan syndrome SHOX-related short stature Turner syndrome

As listed by the trial registrant

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

Noonan syndrome Short stature homeobox- containing gene shox deficiency Turner syndrome

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

    36 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: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • Angers University Hospital Center

    RECRUITING

    Angers, Maine-et-Loire, 49 933, France

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

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Atrium Health Carolinas Medical Center

    RECRUITING

    Charlotte, North Carolina, 28203, United States

  • Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer

    RECRUITING

    Florence, Florence, Firenze, Italy

  • CHU de Toulouse - Hôpital des Enfants

    RECRUITING

    Toulouse, Occitanie, France

  • Central University Hospital of Asturias

    NOT_YET_RECRUITING

    Oviedo, Principality of Asturias, 33011, Spain

  • Centre Hospitalier Universitaire Sainte-Justine

    RECRUITING

    Montreal, Quebec, H3T 1C5, Canada

  • Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Children's Hospital at Montefiore

    RECRUITING

    The Bronx, New York, 10467, United States

  • Children's Hospital of Orange County Main Campus - Orange

    RECRUITING

    Orange, California, 92868, United States

  • Children's Medical Center Dallas

    RECRUITING

    Dallas, Texas, 75235, United States

  • Children's National Medical Center

    RECRUITING

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

  • Childrens Health Queensland Hospital and Health Service

    RECRUITING

    South Brisbane, Queensland, 4101, Australia

  • Childrens Hospital Los Angeles

    RECRUITING

    Los Angeles, California, 90027, United States

  • Childrens Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229-3026, United States

  • Emory University School of Medicine

    RECRUITING

    Atlanta, Georgia, 30322, United States

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    RECRUITING

    Roma, 00 168, Italy

  • Froedtert & Medical College of Wisconsin

    ACTIVE_NOT_RECRUITING

    Milwaukee, Wisconsin, 53226, United States

  • Hospital Clínico Universitario de Santiago

    NOT_YET_RECRUITING

    Santiago de Compostela, La Coruña, 15706, Spain

  • Hôpital Robert-Debré

    RECRUITING

    Paris, Paris, 75019, France

  • Hôpital de la Timone

    RECRUITING

    Marseille, Bouches-du-Rhône, 13385, France

  • IRCCS Istituto Giannina Gaslini

    RECRUITING

    Genova, Genoa, 16147, Italy

  • Icahn School of Medicine at Mount Sinai

    RECRUITING

    New York, New York, 10029, United States

  • Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele

    WITHDRAWN

    Milan, Milan, 20132, Italy

  • Kentucky Children's Hospital

    RECRUITING

    Lexington, Kentucky, 40506, United States

  • M Health Fairview Pediatric Specialty Clinic - Explorer

    WITHDRAWN

    Minneapolis, Minnesota, 55454, United States

  • M.A.G.I.C. Clinic

    NOT_YET_RECRUITING

    Calgary, Alberta, T2E 7H7, Canada

  • Murdoch Children's Research Institute

    RECRUITING

    Parkville, Victoria, 3052, Australia

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

    RECRUITING

    Wilmington, Delaware, 19803, United States

  • New York Medical College

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Nicklaus Children's Hospital

    RECRUITING

    Miami, Florida, 33155, United States

  • Riley Hospital for Children at Indiana University Health

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Royal North Shore Hospital

    RECRUITING

    Saint Leonards, New South Wales, 2065, Australia

  • SickKids - The Hospital for Sick Children

    ACTIVE_NOT_RECRUITING

    Toronto, Ontario, M5G 1X8, Canada

  • South Paris University Hospitals - Bicetre Hospital

    RECRUITING

    Le Kremlin-Bicêtre, Paris, 94270, France

  • St. Luke's Children's Endocrinology and Diabetes

    WITHDRAWN

    Boise, Idaho, 83712, United States

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Texas Health Science Center at Houston (UT Health)

    RECRUITING

    Houston, Texas, 77030, United States

  • Universitätsklinikum des Saarlandes

    RECRUITING

    Homburg, Saarland, 66421, Germany

  • Vanderbilt University Medical Center

    RECRUITING

    Nashville, Tennessee, 37232-2578, United States

More trials for these conditions

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