A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

August 21, 2026 updated by: BioMarin Pharmaceutical

A Phase 2, Randomized, Multicenter, Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

The purpose of this study in children with Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This is a Phase 2, randomized, multicenter, study of vosoritide in children with Noonan syndrome who have inadequate growth during or after human growth hormone (hGH) treatment. The study is intended to characterize the short-term efficacy and safety of 3 dosing regimens of vosoritide. The efficacy and safety of the vosoritide therapeutic dose will be further evaluated, and an analysis of the impact of vosoritide on final adult height (FAH).

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
30

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • Australia
    • New South Wales
      • Saint Leonards, New South Wales, Australia, 2065
    • Queensland
      • South Brisbane, Queensland, Australia, 4101
    • Victoria
      • Parkville, Victoria, Australia, 3052
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2E 7H7
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Active, not recruiting
        • SickKids - The Hospital for Sick Children
    • Quebec
      • Montreal, Quebec, Canada, H3T 1C5
        • Recruiting
        • Centre Hospitalier Universitaire Sainte-Justine
        • Contact:
  • France
    • Bouches-du-Rhône
      • Marseille, Bouches-du-Rhône, France, 13385
    • Maine-et-Loire
      • Angers, Maine-et-Loire, France, 49 933
    • Occitanie
      • Toulouse, Occitanie, France
    • Paris
      • Le Kremlin-Bicêtre, Paris, France, 94270
        • Recruiting
        • South Paris University Hospitals - Bicetre Hospital
        • Contact:
      • Paris, Paris, France, 75019
  • Germany
    • Saarland
      • Homburg, Saarland, Germany, 66421
        • Recruiting
        • Universitatsklinikum des Saarlandes
        • Contact:
  • Italy
    • Florence
      • Florence, Florence, Italy, Firenze
        • Recruiting
        • Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer
        • Contact:
    • Genoa
      • Genova, Genoa, Italy, 16147
    • Milan
      • Milan, Milan, Italy, 20132
        • Withdrawn
        • Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele
  • Spain
    • La Coruña
      • Santiago de Compostela, La Coruña, Spain, 15706
    • Principality of Asturias
      • Oviedo, Principality of Asturias, Spain, 33011
        • Not yet recruiting
        • Central University Hospital of Asturias
        • Contact:
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Childrens Hospital Los Angeles
        • Contact:
      • Orange, California, United States, 92868
        • Recruiting
        • Children's Hospital of Orange County Main Campus - Orange
        • Contact:
    • Colorado
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Recruiting
        • Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
        • Contact:
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
    • Florida
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Recruiting
        • Emory University School of Medicine
        • Contact:
    • Idaho
      • Boise, Idaho, United States, 83712
        • Withdrawn
        • St. Luke's Children's Endocrinology and Diabetes
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Ann & Robert H. Lurie Children's Hospital of Chicago
        • Contact:
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Riley Hospital for Children at Indiana University Health
        • Contact:
    • Iowa
      • Iowa City, Iowa, United States, 52242
    • Kentucky
      • Lexington, Kentucky, United States, 40506
        • Recruiting
        • Kentucky Children's Hospital
        • Contact:
    • Massachusetts
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • Withdrawn
        • M Health Fairview Pediatric Specialty Clinic - Explorer
    • New York
      • New York, New York, United States, 10029
      • The Bronx, New York, United States, 10467
        • Recruiting
        • Children's Hospital at Montefiore
        • Contact:
    • North Carolina
      • Charlotte, North Carolina, United States, 28203
    • Ohio
      • Cincinnati, Ohio, United States, 45229-3026
        • Recruiting
        • Cincinnati Children's Hospital Medical Center
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Childrens Hospital of Philadelphia
        • Contact:
    • Tennessee
      • Nashville, Tennessee, United States, 37232-2578
        • Recruiting
        • Vanderbilt University Medical Center
        • Contact:
    • Texas
      • Dallas, Texas, United States, 75235
      • Houston, Texas, United States, 77030
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Active, not recruiting
        • Froedtert & Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Vosoritide Dose 1 - Low Dose

Drug: Vosoritide - Dose 1 Injection

• Experimental Drug Lyophilized powder for reconstitution
Drug: Vosoritide Injection
Modified recombinant human C-type natriuretic peptide Vosoritide
Experimental: Vosoritide Dose 2 - Medium Dose

Drug: Vosoritide - Dose 2 Injection

• Experimental Drug Lyophilized powder for reconstitution
Drug: Vosoritide Injection
Modified recombinant human C-type natriuretic peptide Vosoritide
Experimental: Vosoritide Dose 3- High Dose

Drug: Vosoritide Dose 3 Injection

• Experimental Drug Lyophilized powder for reconstitution
Drug: Vosoritide Injection
Modified recombinant human C-type natriuretic peptide Vosoritide

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Change from baseline in Annualized Growth Velocity (AGV)
Time Frame: At 6 months
At 6 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Incidence of treatment-emergent adverse events
Time Frame: Until the end of the study, up to 15 years
Until the end of the study, up to 15 years
Change from baseline in height up to Final Adult Height (FAH)
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Change from baseline in height Z-score up to FAH
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Time vosoritide is present at maximum concentration (Tmax)
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Maximum vosoritide observed plasma concentration (Cmax)
Time Frame: Every 6 months through he end of the study, up to15 years
Every 6 months through he end of the study, up to15 years
Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Elimination half-life of vosoritide (t½)
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Apparent clearance of vosoritide (CL/F)
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Apparent volume of distribution of vosoritide (Vz/F)
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Change from baseline in serum collagen X marker (CXM)
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Change from baseline in bone age/chronological age
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
Change from baseline in total body (less head) BMD Z-score
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
Change from baseline in lumbar spine bone mineral density (BMD) Z-score
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
Change from baseline in total body (less head) bone mineral content (BMC)
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
Incidence of new diagnosis of hypertrophic cardiomyopathy in children with Noonan syndrome
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
Incidence of cardiac conditions requiring discontinuation of study treatment
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
12-month interval AGV summarized by age and sex up to FAH
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
Tanner stage over the course of the study
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Change from pre-dose in urine cyclic guanine monophosphate (cGMP)
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Incidence of bone-related events of special interest (fracture, slipped capital femoral epiphysis and avascular necrosis or osteonecrosis)
Time Frame: Throughout study
Throughout study
Change from baseline in height
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Change from baseline in height Z-score
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Change from baseline in 12-month interval AGV
Time Frame: Until the end of the study, up to 15 years
Until the end of the study, up to 15 years
Change from baseline in upper to lower body segment ratio
Time Frame: Until the end of the study, up to 15 years
Until the end of the study, up to 15 years
Change from baseline in arm span to height ratio
Time Frame: Until the end of the study, up to 15 years
Until the end of the study, up to 15 years
Change from baseline in lumbar spine BMC. Change from baseline in lower extremity BMD/BMC [Time Frame: Every 12 months through the end of the study, up to 15 years].
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
Change in the growth plates, long bone growth and bone morphology based on whole length lower extremity X-rays [Time Frame: Every 12 months through the end of the study, up to 15 years]
Time Frame: Every 6 months through the end of the study, up to 15 years
Every 6 months through the end of the study, up to 15 years
Change from baseline in the physical domain score and total score of the QoLISSY
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
Change from baseline in the physical and social domain scores and total score of the PedsQL
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
Change from baseline in PGI-S and CaGI-S item scores
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
PGI-C and CaGI-C item scores
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
Change from baseline in PROMIS-SF Physical Activity score
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years
Change from baseline in KABC-II NVI scores
Time Frame: Every 12 months through the end of the study, up to 15 years
Every 12 months through the end of the study, up to 15 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
BioMarin Pharmaceutical

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, MD, PhD, BioMarin Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 22, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 1, 2041

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 30, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 30, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 31, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 24, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 21, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 111-211

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings