Vosoritide
(111-211 [Canopy NS])
Trial Status Recruiting
Condition
Noonan Syndrome
Phase
- Peptide
Title
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Study Description
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.
Primary Outcome Measure*
- Change from baseline in annualized growth velocity (AGV)
Select Inclusion Criteria*
- Patients ≥ 3 years old, and < 10 years old (females) or < 11 years old (males)
- Height Z-score ≤ -2 standard deviations in reference to the general population of the same age and sex
- Have been receiving continuous human growth hormone (hGH) for the treatment of short stature associated with their condition for a minimum of 1 year immediately prior to enrollment
Secondary Outcome Measures*
- Patients ≥ 3 years old, and < 11 years old (females) or < 12 years old (males)
- Tanner Stage 1
- Previous or current hGH treatment for short stature associated with their condition
Select Exclusion Criteria*
- Patients with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia
- Bone age advanced beyond chronological age by more than 2 years
- 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
* Additional measures/criteria may apply.
For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06668805
Pre-IND
Phase 1
Phase 2
Phase 3
Phase 4