Experimental drug for rare lung disease shows early promise but trial halted

NCT ID NCT02363946

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

Summary

This early-phase study tested a drug called ARC-AAT in healthy volunteers and people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can lead to lung and liver damage. The main goal was to check safety and how the drug affects levels of a key protein in the blood. The trial was terminated early, but results help guide future research.

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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

    Melbourne, Victoria, 3004, Australia

  • Research Site 2

    Homburg, Germany

  • Research Site 3

    Leiden, 2333ZA, Netherlands

  • Research Site 4

    Edgbaston, Birmingham, B15 2WB, United Kingdom