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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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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20-Year quest to map the journey of rare childhood liver diseases
Knowledge-focused TerminatedThis study aims to understand how four rare genetic liver diseases progress over many years in children and young adults. Researchers will follow participants for up to 20 years to track health changes, complications, and outcomes like growth, liver function, and need for transpl…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Arbor Research Collaborative for Health • Aim: Knowledge-focused
Last updated Mar 23, 2026 15:16 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