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

Recruiting now · Phase 2

Conditions studied: Noonan Syndrome

In brief

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.

Key facts

Study ID

NCT06668805

Run by

BioMarin Pharmaceutical

People needed

30

Starts

2024-11-22

Expected to finish

2041-09-01

Last updated by the study team

2026-05-11

Who can join

Age: 3 and older, up to 11. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or 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

You may not qualify if…

• Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia.
• 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.
• 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.

Where it is running

• Childrens Hospital Los Angeles — Los Angeles, California, United States (enrolling)
• Children's Hospital of Orange County Main Campus - Orange — Orange, California, United States (enrolling)
• Children's Hospital Colorado — Aurora, Colorado, United States (enrolling)
• Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children) — Wilmington, Delaware, United States (enrolling)
• Children's National Medical Center — Washington D.C., District of Columbia, United States (enrolling)
• Universitätsklinikum des Saarlandes — Homburg, Saarland, Germany (enrolling)
• Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer — Florence, Florence, Italy (enrolling)
• Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Roma, Italy (enrolling)
• Emory University School of Medicine — Atlanta, Georgia, United States (enrolling)
• St. Luke's Children's Endocrinology and Diabetes — Boise, Idaho, United States (enrolling)
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
• Riley Hospital for Children at Indiana University Health — Indianapolis, Indiana, United States (enrolling)
• University of Iowa — Iowa City, Iowa, United States (enrolling)
• Kentucky Children's Hospital — Lexington, Kentucky, United States (enrolling)
• New York Medical College — Boston, Massachusetts, United States (enrolling)
• M Health Fairview Pediatric Specialty Clinic - Explorer — Minneapolis, Minnesota, United States (enrolling)
• Icahn School of Medicine at Mount Sinai — New York, New York, United States (enrolling)
• Children's Hospital at Montefiore — The Bronx, New York, United States (enrolling)
• Atrium Health Carolinas Medical Center — Charlotte, North Carolina, United States (enrolling)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
• Childrens Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• Vanderbilt University Medical Center — Nashville, Tennessee, United States (enrolling)
• Children's Medical Center Dallas — Dallas, Texas, United States (enrolling)
• University of Texas Health Science Center at Houston (UT Health) — Houston, Texas, United States (enrolling)
• Royal North Shore Hospital — Saint Leonards, New South Wales, Australia (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.