Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants

What Was Tested

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

It had two arms (groups): BMN 111 injection with vial and syringe (active comparator) and BMN 111 injection with injector pen (experimental).

Study treatment will be provided in glass vials. Each glass vial will be labeled as required per country requirement. Pre-filled Diluent Transfer Syringes will be provided for reconstitution.

The registry classes the interventions as combination product and drug.

Who Took Part

36 actual participants took part.

It was open to people of any sex aged from 18 years up to 55 years.

Healthy volunteers could join, not only people with the condition.

Examples of who could join: Participants must be aged 18 to 55 years inclusive, at the time of signing the informed consent. Body weight ≥ 60 kg and \< 110 kg and body mass index within the range 18.0 to 32.0 kg/m2 (inclusive). Males and/or females are eligible to participate in this clinical study. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Females will not be pregnant or lactating, and females of childbearing potential and males will agree to use contraception.

The full record lists 8 inclusion and 23 exclusion criteria; among the exclusions: history of migraines; positive hepatitis panel and/or positive human immunodeficiency virus test; exposure to mycobacterium tuberculosis, indicative of tuberculosis infection, as confirmed by a positive quantiferon-tb gold test.

How the Study Was Run

Design: randomized, crossover and open-label.

The participants were allocated by chance, so the Vosoritide group and the comparison group should start out alike.

Each person switched between Vosoritide and the comparison at some point, usually with a gap in between.

Being open-label, the Vosoritide study did not mask who received what.

It started in 2023-03-15 with completion listed for 2023-06-14.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Maximum observed drug concentration (Cmax) (time frame: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose). Area under the concentration versus time curve from time 0 to infinity (AUC0-∞) (time frame: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose). Area under the concentration versus time curve from time 0 to t, where t is the last timepoint with a measurable concentration (AUC0-t) (time frame: Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose).

It also tracked 4 secondary outcomes, for example Time of Cmax (tmax), Time of t1/2, Time of terminal rate constant (λZ) and Incidence and severity of adverse events.

What This Means in Plain Language

Put simply, this is a phase 1, randomized trial of Vosoritide in 36 people.

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

It ended early (terminated); no results are posted to the registry yet, so nothing here describes outcomes.

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