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A Phase 2, Randomized, Human Growth Hormone-Controlled, Multicenter, Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With an Inadequate Response to Human Growth Hormone. (CANOPY NS, TS, SHOX-D-2)

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Trial Profile

A Phase 2, Randomized, Human Growth Hormone-Controlled, Multicenter, Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With an Inadequate Response to Human Growth Hormone. (CANOPY NS, TS, SHOX-D-2)

Status: Recruiting
Phase of Trial: Phase II

Latest Information Update: 07 Mar 2026

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At a glance

  • Drugs Vosoritide (Primary)
  • Indications Noonan syndrome; Short stature; Turner's syndrome
  • Focus Registrational; Therapeutic Use
  • Acronyms CANOPY NS, TS, SHOX-D-2
  • Sponsors BioMarin Pharmaceutical

Most Recent Events

  • 27 Feb 2026 Number of treatment arms are decreased from 4 to 3 by the removal of Active comparator arm.
  • 27 Feb 2026 Planned number of patients changed from 72 to 54.
  • 04 Aug 2025 According to a BioMarin Pharmaceutical media release, the company expects to share topline data from its pivotal study in the first half of 2026 and file submissions to global health authorities for approval in the second half of 2026, leading to a potential launch in 2027.

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