Experimental drug aims to stop rare heart disease in its tracks

NCT ID NCT03702829

Terminated Disease control Sponsor: Brigham and Women's Hospital Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a drug called inotersen in 31 people with a rare condition where a faulty protein builds up in the heart, causing damage. The drug is designed to lower levels of that protein. The trial was stopped early, so it's unclear if it works, but researchers hoped to see if it could slow the disease.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Inotersen (a drug that lowers a protein called transthyretin made by the liver)
What this could lead to: If successful, this could point toward a treatment that slows or stops heart damage in people with TTR amyloid cardiomyopathy.
What could go wrong: This was a small, early-phase trial that was terminated, so results are limited. The drug may not work or could have side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for AMYLOIDOSIS are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

amyloidosis wild type ATTR amyloidosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

AMYLOIDOSIS

Contacts and locations

Show contact details

Locations

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.