A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

What Was Tested

This registered phase 2 trial tested Vosoritide.

Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery.

It had two arms (groups): Vosoritide + Standard of Care (experimental) and Standard of Care Alone (no intervention).

Institutional standard of care monitoring and treatment for cervicomedullary compression.

The registry classes the interventions as biological.

Who Took Part

20 actual participants took part.

It was open to people of any sex aged from 0 months up to 12 months.

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

Examples of who could join: Parent(s) or guardian(s) willing and able to provide signed informed consent after the nature of the study has been explained and prior to performance of any research related procedure. Have ACH, documented by genetic testing. Are willing and able to perform all study procedures as physically possible. Parent(s) or caregiver(s) are willing to administer daily injections to the subject and complete the required training. Have evidence of CMC that "may" require surgical intervention.

The full record lists 6 inclusion and 8 exclusion criteria; among the exclusions: have hypochondroplasia or short-stature condition other than achondroplasia (eg, trisomy 21, pseudoachondroplasia, etc); have any of the following: untreated congenital hypothyroidism or maternal history of hyperthyroidism, insulin-requiring neonatal diabetes mellitus, autoimmune inflammatory disease, inflammatory bowel disease, autonomic neuropathy; have ever had prior cervicomedullary decompression surgery.

How the Study Was Run

It was run as a randomized, parallel-group and open-label study.

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

Groups ran in parallel: each of the participants stayed in one arm throughout.

Nothing was hidden in this open-label Vosoritide study: the participants and staff both knew the treatment.

It started in 2020-10-10 with completion listed for 2027-12.

It ran at 3 sites in United Kingdom (2) and Australia (1).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: Through Week 260).

It also tracked 1 secondary outcome, for example Evaluate the effect of Vosoritide on total foramen magnum volume (in cm3) by MRI volumetric measurement software.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Vosoritide in 20 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.