Adrenomedullin for CADASIL

What Was Tested

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

Adrenomedullin, a vasoactive peptide, has angiogenic, vasodilation, anti-inflammatory, and anti-oxidative properties and could have triple sites of action on components of the neuro-glial-vascular unit consisting of vessels, microglia and oligodendrocytes or, more specifically, on the white matter oligovascular unit.

It had 1 arm (groups): Adrenomedullin group (experimental).

The registry classes the interventions as drug.

Who Took Part

60 actual participants took part.

It was open to people of any sex aged from 20 years up to 90 years.

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

Examples of who could join: Patients aged between 20 and 90 at the time of obtaining consent. Patients diagnosed as CADASIL after confirming NOTCH3 gene mutation by genetic testing. Patients with Mini-mental state examination-J score of 10-27 or Trail maiking test score (age ajustment) of average + 1.5 SD (standard deviation) or higher.

The full record lists 4 inclusion and 21 exclusion criteria; among the exclusions: patients who cannot perform cognitive function tests (deafness, blindness, etc., mmse-j less than 10 points severe cognitive impairment, etc.); patients received reatment with prohibited drugs or prohibited therapy within the past 12 weeks from the time of registration; patients with active infections requiring antibiotic treatment at registration.

How the Study Was Run

It was run as a single-group and open-label study.

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

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

It started in 2022-01-06 with completion listed for 2023-01-23.

It ran at 1 site in Japan.

Its stated primary purpose is treatment.

What Was Measured

It also tracked 10 secondary outcomes, for example Mean diffusivity change rate of the white matter evaluated by MR diffusion tensor imaging, Fractional anisotropy change rate of the white matter evaluated by MR diffusion tensor imaging, Change in times of Trail making test-A/B from baseline evaluation, Change in scores of Montreal cognitive assessment from baseline evaluation, Change in scores of Wechsler Adult Intelligence Scale-Fourth edition from baseline evaluation and Safety: Serious adverse event.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Adrenomedullin in 60 people.

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

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