A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing … (NCT06668805) | Clinical Trial Compass
RecruitingPhase 2
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
United States54 participantsStarted 2024-11-22
Plain-language 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.
Who can participate
Age range3 Years – 11 Years
SexALL
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Inclusion criteria
✓. 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 criteria
✕. 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,
What they're measuring
1
Change from baseline in Annualized Growth Velocity (AGV)
. 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.