Status:
RECRUITING
A 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
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Short Stature Homeobox- Containing Gene SHOX Deficiency
Noonan Syndrome
Eligibility:
All Genders
3-11 years
Phase:
PHASE2
Brief 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 o...
Detailed Description
This is a Phase 2, randomized, multicenter, basket study of vosoritide in children with Turner syndrome, short stature homeobox-containing gene (SHOX) deficiency, or Noonan syndrome who have inadequat...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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.
Key Trial Info
Start Date :
November 22 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 1 2041
Estimated Enrollment :
54 Patients enrolled
Trial Details
Trial ID
NCT06668805
Start Date
November 22 2024
End Date
September 1 2041
Last Update
February 27 2026
Active Locations (24)
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1
Children's Hospital of Orange County Main Campus - Orange
Orange, California, United States, 92868
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
3
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
Wilmington, Delaware, United States, 19803
4
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010