Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

What Was Tested

In this phase 1, phase 2 trial, researchers set out to test Vosoritide.

Prior to enrollment in the interventional arm of study, subjects will be followed for a minimum of 24 weeks to gather information on safety profiles and determine annualized growth velocity.

The primary study endpoint is the determination of safety and tolerability of daily vosoritide treatment in MPS.

It had 1 arm (groups): Vosoritide (other).

The registry classes the interventions as drug.

More on the Plan

The investigators propose to conduct a single arm phase I/II study of Vosoritide (also called VOXZOGO® and BMN111) in 6 pediatric patients with mucopolysaccaridosis (MPS) types IVA and VI; 3 patients with each disease.

This will be a single center study performed at UCSF Children's Hospital, Oakland, under the direction of Dr.

Paul Harmatz, Professor in Residence in the Department of Pediatric Gastroenterology.

Mucopolysaccharidoses (MPS) are a group of ultra rare genetic lysosomal storage diseases caused by deficiency in various enzymes responsible for the breakdown of glycosaminoglycans (GAGs), leading to progressive accumulations of GAGs in the tissues and organs.

Who Took Part

6 actual participants took part.

It was open to people of any sex aged from 5 years up to 10 years.

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

Examples of who could join: Age \>= 5 years and \< 10 years. Tanner stage 1. Clinical Diagnosis of MPS IVA or VI. MPS IVA (3 patients). MPS VI (3 patients).

The full record lists 14 inclusion and 28 exclusion criteria; among the exclusions: ERT naïve; diagnosis with growth hormone deficiency (defined by igf-1 sds \<-1.0 according to age, gender and tanner stage); hypothyroidism, untreated (tsh \>4.0 mu/l).

How the Study Was Run

Design: single-group and open-label.

Everyone knew when Vosoritide was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

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

It started in 2023-09-25 with completion listed for 2028-12-31.

It ran at 1 site in United States.

Its stated primary purpose is other.

What Was Measured

The main thing measured (primary outcome): Safety and tolerability: Incidence of adverse events while treated with vosoritide (time frame: 48 weeks).

It also tracked 1 secondary outcome, for example Change in height velocity while treated with vosoritide.

What This Means in Plain Language

Put simply, this is a phase 1, phase 2 trial of Vosoritide in 6 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.