ALPHA 1-ANTITRYPSIN DEFICIENCY
Clinical trials for ALPHA 1-ANTITRYPSIN DEFICIENCY explained in plain language.
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Experimental injections target liver damage in rare genetic disorder
Disease control TerminatedThis study aimed to test whether multiple doses of an experimental drug called ARC-AAT could safely reduce the buildup of a harmful protein in the livers of adults with Alpha-1 Antitrypsin Deficiency. Participants would have received seven intravenous injections and undergone two…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC
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20-Year quest to unlock secrets of devastating childhood liver diseases
Knowledge-focused TerminatedThis study aims to track the natural progression of four rare genetic liver diseases that affect children, causing bile flow problems. Researchers will follow participants for up to 20 years to understand how these diseases develop, what complications arise, and what factors lead…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Arbor Research Collaborative for Health • Aim: Knowledge-focused
Last updated Apr 02, 2026 14:57 UTC
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Massive biobank launched to unlock secrets of rare genetic disease
Knowledge-focused ENROLLING_BY_INVITATIONThis study is creating a large collection of medical information and tissue samples (like blood, liver, or lung tissue) from people with Alpha-1 Antitrypsin Deficiency. The goal is to build a resource for future scientists to study this genetic condition. Participants provide inf…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University of Florida • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:35 UTC
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Scientists hunt for early warning signs in rare genetic lung disease
Knowledge-focused OngoingThis study aims to find early signs that predict which people with Alpha-1 antitrypsin deficiency, a genetic disease that can damage the lungs and liver, will get worse quickly. Researchers are following 286 adults with the disease for three years, using CT scans and blood tests …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated Mar 02, 2026 15:28 UTC