Noonan SyndromePhase 2NCT06668805
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
A Phase 2 study testing Vosoritide Injection in people with Noonan Syndrome, run by BioMarin Pharmaceutical at 38 sites in 7 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Noonan Syndrome
- What is being tested?
- Vosoritide Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 38 sitesin 7 countries
- Study dates
- 22 Nov 2024to Sep 2041
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
- A clinical diagnosis of Noonan syndrome.
- 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.
- Tanner Stage 1, at time of signing the ICF.
- Previous or current hGH treatment for short stature associated with their condition.
- 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
Who cannot join
- 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.
- Bone age advanced beyond chronological age by more than 2 years.
- Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension,
- Have an unstable condition likely to require surgical intervention during the study.
- 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.
- Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period.
- Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.
Trial locations and contacts
38 sites in 7 countries.
38 sites in 7 countries
United States21 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
New York Medical College
Boston, Massachusetts, 02115
Atrium Health Carolinas Medical Center
Charlotte, North Carolina, 28203
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3026
Children's Medical Center Dallas
Dallas, Texas, 75235
University of Texas Health Science Center at Houston (UT Health)
Houston, Texas, 77030
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202
University of Iowa
Iowa City, Iowa, 52242
Kentucky Children's Hospital
Lexington, Kentucky, 40506
Childrens Hospital Los Angeles
Los Angeles, California, 90027
Nicklaus Children's Hospital
Miami, Florida, 33155
Froedtert & Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-2578
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Children's Hospital of Orange County Main Campus - Orange
Orange, California, 92868
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital at Montefiore
The Bronx, New York, 10467
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
Wilmington, Delaware, 19803
France5 sites
Angers University Hospital Center
Angers, Maine-et-Loire, 49 933
South Paris University Hospitals - Bicetre Hospital
Le Kremlin-Bicêtre, Paris, 94270
Hôpital de la Timone
Marseille, Bouches-du-Rhône, 13385
Hôpital Robert-Debré
Paris, Paris, 75019
CHU de Toulouse - Hôpital des Enfants
Toulouse, Occitanie
Australia3 sites
Murdoch Children's Research Institute
Parkville, Victoria, 3052
Royal North Shore Hospital
Saint Leonards, New South Wales, 2065
Childrens Health Queensland Hospital and Health Service
South Brisbane, Queensland, 4101
Canada3 sites
M.A.G.I.C. Clinic
Calgary, Alberta, T2E 7H7
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, H3T 1C5
SickKids - The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
Italy3 sites
Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer
Florence, Florence, Firenze
IRCCS Istituto Giannina Gaslini
Genova, Genoa, 16147
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 00 168
Spain2 sites
Central University Hospital of Asturias
Oviedo, Principality of Asturias, 33011
Hospital Clínico Universitario de Santiago
Santiago de Compostela, La Coruña, 15706
About this study
Official title: 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.
Read the 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).
- Conditions
- Noonan Syndrome
- Being tested
- Vosoritide Injection
- Keywords
- Short Stature, Musculoskeletal Diseases, Bone Diseases, Developmental Endocrine System Diseases Natriuretic Peptide, C-Type
- Medical subject headings
- Noonan Syndrome, Dwarfism, Musculoskeletal Diseases, Bone Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vosoritide InjectionDrug
Modified recombinant human C-type natriuretic peptide Vosoritide
| Study group | Role | What they receive |
|---|---|---|
| Vosoritide Dose 1 - Low Dose | Experimental | Drug: Vosoritide - Dose 1 Injection
• Experimental Drug Lyophilized powder for reconstitution Receives: Drug: Vosoritide Injection |
| Vosoritide Dose 2 - Medium Dose | Experimental | Drug: Vosoritide - Dose 2 Injection
• Experimental Drug Lyophilized powder for reconstitution Receives: Drug: Vosoritide Injection |
| Vosoritide Dose 3- High Dose | Experimental | Drug: Vosoritide Dose 3 Injection
• Experimental Drug Lyophilized powder for reconstitution Receives: Drug: Vosoritide Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Annualized Growth Velocity (AGV)
Measured: At 6 months
Secondary outcomes (34)
Incidence of treatment-emergent adverse events
Measured: Until the end of the study, up to 15 years
Incidence of new diagnosis of hypertrophic cardiomyopathy in children with Noonan syndrome
Measured: Every 12 months through the end of the study, up to 15 years
Incidence of cardiac conditions requiring discontinuation of study treatment
Measured: Every 12 months through the end of the study, up to 15 years
Change from baseline in height
Measured: Every 6 months through the end of the study, up to 15 years
Change from baseline in height Z-score
Measured: Every 6 months through the end of the study, up to 15 years
Change from baseline in 12-month interval AGV
Measured: Until the end of the study, up to 15 years
Change from baseline in upper to lower body segment ratio
Measured: Until the end of the study, up to 15 years
Change from baseline in arm span to height ratio
Measured: Until the end of the study, up to 15 years
Change from baseline in height up to Final Adult Height (FAH)
Measured: Every 6 months through the end of the study, up to 15 years
Change from baseline in height Z-score up to FAH
Measured: Every 6 months through the end of the study, up to 15 years
12-month interval AGV summarized by age and sex up to FAH
Measured: Every 12 months through the end of the study, up to 15 years
Tanner stage over the course of the study
Measured: Every 6 months through the end of the study, up to 15 years
Time vosoritide is present at maximum concentration (Tmax)
Measured: Every 6 months through the end of the study, up to 15 years
Maximum vosoritide observed plasma concentration (Cmax)
Measured: 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)
Measured: 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-∞)
Measured: Every 6 months through the end of the study, up to 15 years
Elimination half-life of vosoritide (t½)
Measured: Every 6 months through the end of the study, up to 15 years
Apparent clearance of vosoritide (CL/F)
Measured: Every 6 months through the end of the study, up to 15 years
Apparent volume of distribution of vosoritide (Vz/F)
Measured: Every 6 months through the end of the study, up to 15 years
Change from pre-dose in urine cyclic guanine monophosphate (cGMP)
Measured: Every 6 months through the end of the study, up to 15 years
Change from baseline in serum collagen X marker (CXM)
Measured: Every 6 months through the end of the study, up to 15 years
Change from baseline in bone age/chronological age
Measured: Every 12 months through the end of the study, up to 15 years
Change from baseline in total body (less head) BMD Z-score
Measured: 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
Measured: 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)
Measured: Every 12 months through 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].
Measured: 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]
Measured: 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)
Measured: Throughout study
Change from baseline in the physical domain score and total score of the QoLISSY
Measured: 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
Measured: Every 12 months through the end of the study, up to 15 years
Change from baseline in PGI-S and CaGI-S item scores
Measured: Every 12 months through the end of the study, up to 15 years
PGI-C and CaGI-C item scores
Measured: Every 12 months through the end of the study, up to 15 years
Change from baseline in PROMIS-SF Physical Activity score
Measured: Every 12 months through the end of the study, up to 15 years
Change from baseline in KABC-II NVI scores
Measured: Every 12 months through the end of the study, up to 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2024
- Study start22 Nov 2024
- Primary completionMar 2027 (estimated)
- Study completionSep 2041 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Noonan SyndromeStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, PhD, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06668805
- Sponsor's protocol ID
- 111-211
- Status last verified
- Sep 2026
- First submitted
- 30 Oct 2024
- Last updated
- 23 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Multicenter, Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
- ClinicalTrials.gov ID
- NCT06668805
- Acronym / sponsor ID
- 111-211
- Conditions
- Noonan Syndrome
- Keywords
- Short Stature, Musculoskeletal Diseases, Bone Diseases, Developmental Endocrine System Diseases Natriuretic Peptide, C-Type
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 22 Nov 2024 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Sep 2041 (estimated)
- First posted
- 31 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Vosoritide Dose 1 - Low Dose | Experimental | Drug: Vosoritide InjectionDrug: Vosoritide - Dose 1 Injection • Experimental Drug Lyophilized powder for reconstitution |
| Vosoritide Dose 2 - Medium Dose | Experimental | Drug: Vosoritide InjectionDrug: Vosoritide - Dose 2 Injection • Experimental Drug Lyophilized powder for reconstitution |
| Vosoritide Dose 3- High Dose | Experimental | Drug: Vosoritide InjectionDrug: Vosoritide Dose 3 Injection • Experimental Drug Lyophilized powder for reconstitution |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
- A clinical diagnosis of Noonan syndrome.
- 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.
- Tanner Stage 1, at time of signing the ICF.
- Previous or current hGH treatment for short stature associated with their condition.
Key exclusion criteria (7 listed)
- 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.
- Bone age advanced beyond chronological age by more than 2 years.
- Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension,
- Have an unstable condition likely to require surgical intervention during the study.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vosoritide InjectionModified recombinant human C-type natriuretic peptide Vosoritide | Drug | Vosoritide Dose 1 - Low Dose, Vosoritide Dose 2 - Medium Dose, Vosoritide Dose 3- High Dose |
Primary outcome
Change from baseline in Annualized Growth Velocity (AGV)
Time frame: At 6 months
34 secondary outcomes
Incidence of treatment-emergent adverse events
Time frame: Until 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
Incidence of cardiac conditions requiring discontinuation of study treatment
Time frame: Every 12 months through the end of the study, up to 15 years
Change from baseline in height
Time frame: 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
Change from baseline in 12-month interval AGV
Time frame: 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
Change from baseline in arm span to height ratio
Time frame: 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
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
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
Tanner stage over the course of the study
Time frame: 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
Maximum vosoritide observed plasma concentration (Cmax)
Time frame: 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
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
Elimination half-life of vosoritide (t½)
Time frame: 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
Apparent volume of distribution of vosoritide (Vz/F)
Time frame: 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
Change from baseline in serum collagen X marker (CXM)
Time frame: 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
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
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
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
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
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
Incidence of bone-related events of special interest (fracture, slipped capital femoral epiphysis and avascular necrosis or osteonecrosis)
Time frame: Throughout study
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
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
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
PGI-C and CaGI-C item scores
Time frame: 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
Change from baseline in KABC-II NVI scores
Time frame: Every 12 months through the end of the study, up to 15 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director, MD, PhD study director
BioMarin Pharmaceutical
38 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vosoritide Injection in people with Noonan Syndrome, run by BioMarin Pharmaceutical at 38 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 23 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 38 sites in United States, France, Australia, Canada, Italy and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in Annualized Growth Velocity (AGV) (time frame: At 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Sep 2026.