New shot aims to stop liver damage in rare genetic disease
NCT ID NCT03945292
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This Phase 2 study tested a drug called fazirsiran in 40 adults with Alpha-1 antitrypsin deficiency, a genetic condition that can cause liver disease. The drug is designed to lower levels of a harmful protein (Z-AAT) in the blood. Researchers measured safety and how much the protein dropped after 16 weeks of treatment.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- fazirsiran (also called TAK-999 or ARO-AAT), given as a shot under the skin
- What this could lead to
- If it works, this could lead to a treatment that lowers the toxic protein buildup in the liver, potentially slowing or stopping liver damage in people with Alpha-1 antitrypsin deficiency.
- What could go wrong
- This is an early (Phase 2) trial with only 40 participants, so results may not apply to everyone. The drug may not work as hoped, and side effects are still being studied.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Charleston, South Carolina, 29425, United States
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Research Site 10
St Louis, Missouri, 63104, United States
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Research Site 11
Sacramento, California, 95817, United States
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Research Site 12
New York, New York, 10032, United States
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Research Site 13
Stanford, California, 94305, United States
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Research Site 14
La Jolla, California, 92037, United States
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Research Site 15
San Francisco, California, 94158, United States
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Research Site 16
Salt Lake City, Utah, 84132, United States
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Research Site 17
Los Angeles, California, 90095, United States
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Research Site 19
Houston, Texas, 77030, United States
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Research Site 2
Gainesville, Florida, 32610, United States
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Research Site 20
Barcelona, 08035, Spain
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Research Site 22
Pavia, 27100, Italy
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Research Site 23
Leiden, 2333 ZA, Netherlands
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Research Site 24
Funchal, 9004-514, Portugal
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Research Site 27
Aachen, 52074, Germany
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Research Site 3
Iowa City, Iowa, 52242, United States
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Research Site 4
Nashville, Tennessee, 37232, United States
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Research Site 5
Birmingham, Alabama, 35233, United States
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Research Site 7
Indianapolis, Indiana, 46202, United States
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Research Site 9
Phoenix, Arizona, 85054, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Could a simple shot replace IV drips for Alpha-1 patients?
- Hidden liver harm: study tracks silent damage in genetic disorder
- Promising liver drug trial halted early: what it means for patients
- 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