Actively Recruiting
A Phase 2, Randomized, Multicenter Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Led by BioMarin Pharmaceutical · Updated on 2026-05-11
30
Participants Needed
36
Research Sites
756 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying children with Noonan syndrome who have inadequate growth during or after treatment with human growth hormone (hGH). The study aims to evaluate the effects of three different doses of vosoritide on growth, specifically looking at annualized growth velocity (AGV) after six months of treatment. The long-term safety and effectiveness of vosoritide at the therapeutic dose will be assessed up to when participants reach their final adult height (FAH). This Phase 2, randomized, multicenter trial compares three dosing regimens of vosoritide, which is given as an injection. Participants receive one of three different doses of this experimental drug, provided as a lyophilized powder for reconstitution. The study includes both a short-term evaluation of growth changes over six months and a longer-term assessment of height and safety measures up to 15 years. During the study, children will have regular height measurements and assessments of growth velocity, body proportions, bone density, and bone age. Researchers will also monitor heart conditions, bone-related events, and quality of life using various questionnaires and physical activity scores. Blood and urine tests will track vosoritide levels and biological markers. Safety and growth will be followed every six to twelve months until participants reach their final adult height, which may take up to 15 years.
CONDITIONS
Brief Title
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be 3 years old or older, and under 11 years old for females or under 12 years old for males, at the time of consent.
- Genetically confirmed diagnosis of Turner syndrome, SHOX deficiency, or Noonan syndrome.
- Height corresponding to a Z-score of -1.28 or lower (below the 10th percentile) for age and sex.
- Tanner Stage 1 at the time of consent.
- Previous or current treatment with human growth hormone for short stature related to their condition.
- Inadequate growth confirmed by an annualized growth velocity below the average for their age and sex.
You will not qualify if you...
- Turner syndrome participants with Y-chromosome material unless they have had gonadectomy and have fully external female genitalia.
- Diagnosis of other systemic diseases that may cause short stature besides Turner syndrome, SHOX deficiency, or Noonan syndrome.
- Bone age advanced more than 2 years beyond chronological age.
- Uncorrected congenital heart disease that increases risk during low blood pressure.
- Unstable condition likely to need surgical intervention during the study.
- Evidence of decreased growth velocity less than 1.5 cm/year over at least 6 months and growth plate closure.
- Previous, planned, or expected limb-lengthening surgery during the study.
- Planned or expected bone-related surgery during the study, excluding tooth extraction.
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 15 years
Participants receive one of three dosing regimens of vosoritide to evaluate its short-term efficacy and safety for growth improvement.
Regular visits every 6 months for dosing and assessments
Duration - Up to 15 years
Participants are monitored for long-term safety and growth outcomes after treatment ends.
Annual visits for ongoing safety and efficacy assessments
Trial Site Locations
Total: 36 locations
1
Childrens Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
2
Children's Hospital of Orange County Main Campus - Orange
Orange, California, United States, 92868
Actively Recruiting
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
4
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
Wilmington, Delaware, United States, 19803
Actively Recruiting
5
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
6
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Not Yet Recruiting
7
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Actively Recruiting
8
St. Luke's Children's Endocrinology and Diabetes
Boise, Idaho, United States, 83712
Actively Recruiting
9
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
10
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, United States, 46202
Actively Recruiting
11
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
12
Kentucky Children's Hospital
Lexington, Kentucky, United States, 40506
Actively Recruiting
13
New York Medical College
Boston, Massachusetts, United States, 02115
Actively Recruiting
14
M Health Fairview Pediatric Specialty Clinic - Explorer
Minneapolis, Minnesota, United States, 55454
Actively Recruiting
15
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029
Actively Recruiting
16
Children's Hospital at Montefiore
The Bronx, New York, United States, 10467
Actively Recruiting
17
Atrium Health Carolinas Medical Center
Charlotte, North Carolina, United States, 28203
Actively Recruiting
18
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3026
Actively Recruiting
19
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
20
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232-2578
Actively Recruiting
21
Children's Medical Center Dallas
Dallas, Texas, United States, 75235
Actively Recruiting
22
University of Texas Health Science Center at Houston (UT Health)
Houston, Texas, United States, 77030
Actively Recruiting
23
Royal North Shore Hospital
Saint Leonards, New South Wales, Australia, 2065
Actively Recruiting
24
Childrens Health Queensland Hospital and Health Service
South Brisbane, Queensland, Australia, 4101
Actively Recruiting
25
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
Actively Recruiting
26
M.A.G.I.C. Clinic
Calgary, Alberta, Canada, T2E 7H7
Actively Recruiting
27
Hôpital de la Timone
Marseille, Bouches-du-Rhône, France, 13385
Actively Recruiting
28
Angers University Hospital Center
Angers, Maine-et-Loire, France, 49 933
Actively Recruiting
29
CHU de Toulouse - Hôpital des Enfants
Toulouse, Occitanie, France
Actively Recruiting
30
South Paris University Hospitals - Bicetre Hospital
Le Kremlin-Bicêtre, Paris, France, 94270
Actively Recruiting
31
Hôpital Robert-Debré
Paris, Paris, France, 75019
Actively Recruiting
32
Universitätsklinikum des Saarlandes
Homburg, Saarland, Germany, 66421
Actively Recruiting
33
Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer
Florence, Florence, Italy, Firenze
Actively Recruiting
34
IRCCS Istituto Giannina Gaslini
Genova, Genoa, Italy, 16147
Not Yet Recruiting
35
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, Italy, 00 168
Actively Recruiting
36
Central University Hospital of Asturias
Oviedo, Principality of Asturias, Spain, 33011
Not Yet Recruiting
Research Team
T
Trial Specialist
S
Study Manager
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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