A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers

What Was Tested

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

Information about any side effects that may occur will also be collected.

It had two arms (groups): Placebo (placebo comparator) and BMN 111 (active comparator).

The registry classes the interventions as drug.

Who Took Part

Enrollment: 74 planned participants were the target.

It was open to men only aged from 22 years up to 45 years.

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

Examples of who could join: Is willing and able to provide written, signed informed consent (legally authorized representative) after the nature of the study has been explained and prior to performance of any research-related procedure. Is a male 22 to 45 years of age, inclusive. Has a body weight between 63 and 100 kg, inclusive. Has a body mass index (BMI) between 18 and 32 kg/m2, inclusive. Is in good health generally, as determined by medical history, physical examination, clinical laboratory evaluations, and 12-lead electrocardiogram (ECG) at Screening.

The full record lists 8 inclusion and 26 exclusion criteria; among the exclusions: baseline systolic blood pressure \< 100 mmhg; body weight \[kg\] / 72 serum creatinine \[mg/dl\]; has anemia (hb \< 12.5 gm/dl).

How the Study Was Run

Design: randomized, factorial and double-blind.

Random assignment meant chance, not the researchers, decided who got Vosoritide and who got the comparison, which limits bias.

Neither the participants nor the staff measuring them knew who was on Vosoritide, so expectations could not easily colour the readings.

Those kept unaware of assignments: participant and investigator.

It started in 2012-02 with completion listed for 2012-06.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Safety based on vitals signs. Safety based on adverse events.

It also tracked 2 secondary outcomes, for example Safety based on cardiovascular effects.

What This Means in Plain Language

Put simply, this is a phase 1, randomized trial of Vosoritide in 74 people.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

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