Vosoritide

(111-210 [Canopy ISS-2])

Trial Status Recruiting

Condition

Idiopathic Short Stature

Technology

• Peptide

Title

A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Study Description

This study will evaluate the effect of multiple doses of vosoritide and the effect of vosoritide compared to human growth hormone (hGH), in children with Idiopathic Short Stature.

Primary Outcome Measures*

• Change in annualized growth velocity (AGV), height, and height Z-score

Secondary Outcome Measures*

• Incidence of adverse events
• Change in pharmacodynamic parameters (urine cyclic guanine monophosphate, serum collagen X marker)
• Pharmacokinetics of vosoritide

Select Inclusion Criteria*

• Patients ≥3 and <10 (female) or < 11 (males) years of age
• Height Z-score ≤-2.25 standard deviations compared to age and sex matched population norms
• If ≥ 5 years at Screening, must be Tanner Stage I

Select Exclusion Criteria*

• Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to: Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome), ACAN deficiency
• History of treatment with a growth promoting agent

* Additional measures/criteria may apply.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06382155

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Idiopathic Short Stature

(111-903 [Canopy ISS-OS])

Trial Status Recruiting

Condition

Idiopathic Short Stature

Technology

• Observational

Title

A Study to Assess Growth in Children With Idiopathic Short Stature

Study Description

This study will collect growth measurements and other variables of interest to generate baseline growth data in children with Idiopathic Short Stature.

Primary Outcome Measures*

• Change in growth (annualized growth velocity, height Z-score, height) and anthropometric measures (body mass index [BMI], BMI Z-score)

Secondary Outcome Measures*

• Rates of medical events of interest
• Association between specific variants and growth velocity

Select Inclusion Criteria*

• Patients > 2 and ≤ 14 (female) or ≤ 16 (males) years of age
• Height Z-score ≤-2.5 standard deviations compared to age and sex matched population norms

Select Exclusion Criteria*

• Presence of pituitary hormone deficiencies
• Bone age advanced over chronological age by >3 years
• Received an investigational product or medical device within last 6 months

* Additional measures/criteria may apply.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06309979

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4