Experimental drug for rare lung disease shows early promise but trial halted
NCT ID NCT02363946
First seen Jun 27, 2026 · 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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Research Site 1
Melbourne, Victoria, 3004, Australia
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Research Site 2
Homburg, Germany
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Research Site 3
Leiden, 2333ZA, Netherlands
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Research Site 4
Edgbaston, Birmingham, B15 2WB, United Kingdom
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Hidden liver harm: study tracks silent damage in genetic disorder
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- Gene-Editing shot aims to fix lung and liver damage in rare disease
- New drug AIR-001 enters first human tests for rare lung condition