Liver disease drug trial pulled before it even started

NCT ID NCT02900183

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

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study aimed to test an experimental drug called ARC-AAT in people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can cause liver and lung damage. The drug was designed to reduce harmful protein buildup in the liver. However, the trial was withdrawn before any participants were enrolled, so no data on safety or effectiveness were collected.

What this could mean

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

Active substance: ARC-AAT (an RNA interference drug)
What this could lead to: If it had worked, this could have pointed toward a treatment to reduce harmful protein buildup in the liver for people with Alpha-1 Antitrypsin Deficiency.
What could go wrong: This trial was withdrawn before enrolling anyone, so no results are available. The approach was still early (Phase 2) and required invasive liver biopsies, which carry risks.

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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

Locations

Research Site 1

Toronto, Ontario, M5T3A9, Canada
Research Site 2

Dublin, Ireland
Research Site 3

Pavia, 27100, Italy
Research Site 4

Malmö, SE-205 02, Sweden

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