A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone

Study Details

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)

Conditions:

Short Stature Homeobox- Containing Gene SHOX Deficiency, Noonan SyndromeTurner Syndrome.

Study ID

GEN24012 (BMN 111-211)

Study Description

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 versus hGH 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.

Eligibility Requirements

Inclusion Criteria

  1. 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
  2. A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome.
  3. A height assessment corresponding to a height Z-score of > -2.00 SDs and ≤ -1.75 SDs (up to 20% of participants)/≤ -2.00 SDs (at least 80% of participants) in reference to the general population of the same age and sex.
  4. Tanner Stage 1, at time of signing the ICF.
  5. Have been receiving continuous hGH for the treatment of short stature associated with their condition for a minimum of 1 year immediately prior to enrollment and be receiving a dose of ≥ 0.35 mg/kg weekly, with no weight-based dosing changes in the last 6 months and none planned in the future.
  6. Are willing to continue on hGH at their current dose for the Baseline Growth Phase, and for 2 years post randomization if randomized to the hGH arm.
  7. Inadequate response to prior hGH treatment.

Exclusion Criteria

  1. Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia.
  2. 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.
  3. Bone age advanced beyond chronological age by more than 2 years.
  4. Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension,
  5. Have an unstable condition likely to require surgical intervention during the study.
  6. 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.
  7. Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period.
  8. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.

Principal Investigator

Ana Maria Rodriguez Barreto, MD

Clinical Geneticist

More Information


This page was last updated on: 4/28/2026 3:24:31 PM