Liver disease drug trial pulled before it even started

NCT ID NCT02900183

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 However, the trial was withdrawn before any participants were enrolled, so no data on safety or effectiveness were collected.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This trial was withdrawn before enrolling anyone, so no results are available.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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.

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