A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

What Was Tested

The trial (phase 2) is built around Vosoritide.

It had three arms (groups): one (experimental), one (experimental) and one (experimental).

The registry classes the interventions as drug.

More on the Plan

This is a Phase 2, randomized, multicenter, study of vosoritide in children with Noonan syndrome who have inadequate growth during or after human growth hormone (hGH) treatment.

Who Took Part

30 planned participants were the target.

It was open to people of any sex aged from 3 years up to 11 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: 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 clinical diagnosis of 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. 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.

The full record lists 6 inclusion and 7 exclusion criteria; among the exclusions: diagnosis of systemic disease or condition that may cause short stature other than noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease; bone age advanced beyond chronological age by more than 2 years; have an unstable condition likely to require surgical intervention during the study.

How the Study Was Run

It was run as a randomized, parallel-group and triple-blind study.

Because people in this Vosoritide study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

The Vosoritide group and the comparison group ran side by side rather than switching.

As a double-blind Vosoritide study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant, care provider and investigator.

It started in 2024-11-22 with completion listed for 2027-03.

It ran at 38 sites in United States (21), France (5), Australia (3), Canada (3), Italy (3) and Spain (2).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Change from baseline in Annualized Growth Velocity (AGV) (time frame: At 6 months).

It also tracked 34 secondary outcomes, for example Incidence of treatment-emergent adverse events, Incidence of new diagnosis of hypertrophic cardiomyopathy in children with Noonan syndrome, Incidence of cardiac conditions requiring discontinuation of study treatment, Change from baseline in height, Change from baseline in height Z-score and Change from baseline in 12-month interval AGV.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Vosoritide in 30 people.

Its size is small, so on its own it can only give an early look.

Its status is active not recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Vosoritide compound page, all human trials.