Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

What Was Tested

In this phase 3 trial, researchers set out to test Vosoritide.

The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).

It had two arms (groups): vosoritide injection with vial and syringe (experimental) and Placebo injection with vial and syringe (placebo comparator).

The registry classes the interventions as drug.

More on the Plan

This is a Phase 3 randomized, stratified, placebo-controlled, double-blind multicenter study to evaluate the effect of 52 weeks of daily vosoritide administration on annualized growth velocity (AGV) in participants with HCH.

Eligible participants with documented HCH confirmed by genetic testing will roll over from Study 111-902 and enter the 111-303 study.

Participants will be randomly assigned to one of two treatment groups: Placebo or Vosoritide.

The route of administration is subcutaneous injection, and the frequency of administration is daily.

Who Took Part

The registry lists 80 planned participants were the target.

It was open to people of any sex aged from 3 years up to 17 years.

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

Examples of who could join: Participants must be ≥ 3 to \< 18 years of age at enrollment. A confirmed genetic diagnosis of HCH. Males and females are eligible to participate in this clinical study.

The full record lists 6 inclusion and 13 exclusion criteria; among the exclusions: short stature condition other than hch; have an unstable condition likely to require surgical intervention during the study; taking any of the prohibited medications.

How the Study Was Run

It was run as a randomized, single-group and quadruple-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.

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

Everyone received the same intervention, so there was no separate comparison group inside the trial.

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

It started in 2024-06-17 with completion listed for 2026-08-01.

It ran at 23 sites in United States (4), France (4), Japan (4), Canada (3), Italy (3) and Germany (2).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo (time frame: At week 52).

It also tracked 2 secondary outcomes, for example Change from baseline in standing height at Week 52 versus placebo and Change from baseline in height Z-score at Week 52 versus placebo.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Vosoritide in 80 people.

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.