DRUG
Vosoritide Injection
Modified recombinant human C-type natriuretic peptide Vosoritide
Status
Recruiting
Phase
Phase 2
Enrollment
30
Locations
36
Results
Not posted
Publications
0
Study 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.
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).
Interventions
DRUG
Modified recombinant human C-type natriuretic peptide Vosoritide
Timeline
First posted
Oct 31, 2024
Study start
Nov 22, 2024
Primary completion
Mar 2027
Study completion
Sep 2041
Results posted
Not reported
Registry updated
May 11, 2026
Outcomes
Change from baseline in Annualized Growth Velocity (AGV)
Time frame · At 6 months
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
Eligibility
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 genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or 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. Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia. 2. Diagnosis of systemic disease or condition that may cause short stature other than Turner syndrome, SHOX deficiency, or Noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease. 3. Bone age advanced beyond chronological age by more than 2 years. 4. Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension, 5. Have an unstable condition likely to require surgical intervention during the study. 6. 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. 7. Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period. 8. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.
Study locations
Australia · Canada · France · Germany · Italy · Spain · United States. Showing up to 24 locations stored in the fast local snapshot.
Childrens Hospital Los Angeles
Los Angeles, California, United States
Children's Hospital of Orange County Main Campus - Orange
Orange, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
Wilmington, Delaware, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Emory University School of Medicine
Atlanta, Georgia, United States
St. Luke's Children's Endocrinology and Diabetes
Boise, Idaho, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, United States
University of Iowa
Iowa City, Iowa, United States
Kentucky Children's Hospital
Lexington, Kentucky, United States
New York Medical College
Boston, Massachusetts, United States
M Health Fairview Pediatric Specialty Clinic - Explorer
Minneapolis, Minnesota, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Children's Hospital at Montefiore
The Bronx, New York, United States
Atrium Health Carolinas Medical Center
Charlotte, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Children's Medical Center Dallas
Dallas, Texas, United States
University of Texas Health Science Center at Houston (UT Health)
Houston, Texas, United States
Royal North Shore Hospital
Saint Leonards, New South Wales, Australia
Childrens Health Queensland Hospital and Health Service
South Brisbane, Queensland, Australia
Publications
No PMID-linked publications were present in this registry snapshot.
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