ALPHA 1-ANTITRYPSIN DEFICIENCY
Clinical trials for ALPHA 1-ANTITRYPSIN DEFICIENCY explained in plain language.
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Early drug test aims to tackle root cause of rare lung and liver disease
Disease control TerminatedThis early-stage study tested a new drug called ARC-AAT in healthy volunteers and people with Alpha-1 Antitrypsin Deficiency (AATD). The main goal was to check if the drug was safe and to see if a single dose could lower the levels of a harmful protein in the blood that causes lu…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated Apr 03, 2026 14:42 UTC
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Experimental drug tested for rare genetic lung disease
Disease control TerminatedThis study tested whether the drug VX-864 could help people with a specific genetic form of Alpha-1 antitrypsin deficiency. Researchers enrolled 14 participants with the PiZZ genotype to receive the drug for 48 weeks. The trial was terminated early, and researchers measured chang…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Mar 25, 2026 14:08 UTC