New drug targets liver damage in rare genetic disease

NCT ID NCT03946449

Completed Disease control Sponsor: Arrowhead Pharmaceuticals Source: ClinicalTrials.gov ↗

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

Summary

This study tested a drug called fazirsiran in 16 people with alpha-1 antitrypsin deficiency, a genetic condition that can cause liver damage. The drug aims to reduce harmful protein buildup in the liver. Researchers measured changes in liver protein levels and safety over 24 to 48 weeks.

What this could mean

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

Active substance: fazirsiran (also called TAK-999 or ARO-AAT)
What this could lead to: If it works, this could point toward a treatment that reduces harmful protein buildup in the liver for people with alpha-1 antitrypsin deficiency.
What could go wrong: This is a small, early-phase trial with only 16 participants, so results may not apply to everyone. The drug may not show enough benefit 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 ALPHA 1 ANTITRYPSIN DEFICIENCY are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

alpha 1-antitrypsin deficiency

As listed by the trial registrant

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

ALPHA 1 ANTITRYPSIN DEFICIENCY

Contacts and locations

Show contact details

Locations

Research Center 1

Vienna, 1090, Austria
Research Center 1

Aachen, 52074, Germany
Research Center 2

Edinburgh, EH19 3BJ, United Kingdom
Research Center 3

Cambridge, CB2 0QQ, United Kingdom

More trials for these conditions

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