A Phase 2, Randomized, Multicenter, Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment.
Status: Recruiting
Location: See all (24) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
The purpose of this basket study in children with Turner syndrome, SHOX deficiency, and 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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Maximum Age: 11
Healthy Volunteers: f
View:
• 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
Locations
United States
California
Children's Hospital of Orange County Main Campus - Orange
RECRUITING
Orange
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Washington, D.c.
Children's National Medical Center
RECRUITING
Washington D.c.
Delaware
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
RECRUITING
Wilmington
Florida
Nicklaus Children's Hospital
NOT_YET_RECRUITING
Miami
Idaho
St. Luke's Children's Endocrinology and Diabetes
RECRUITING
Boise
Kentucky
Kentucky Children's Hospital
RECRUITING
Lexington
Massachusetts
New York Medical College
RECRUITING
Boston
Minnesota
M Health Fairview Pediatric Specialty Clinic - Explorer
RECRUITING
Minneapolis
North Carolina
Atrium Health Carolinas Medical Center
RECRUITING
Charlotte
New York
Icahn School of Medicine at Mount Sinai
RECRUITING
New York
Children's Hospital at Montefiore
RECRUITING
The Bronx
Tennessee
Vanderbilt University Medical Center
RECRUITING
Nashville
Texas
Children's Medical Center Dallas
RECRUITING
Dallas
University of Texas Health Science Center at Houston (UT Health)
RECRUITING
Houston
Other Locations
Australia
Murdoch Children's Research Institute
RECRUITING
Parkville
France
Angers University Hospital Center
RECRUITING
Angers
South Paris University Hospitals - Bicetre Hospital