One-Shot gene therapy aims to stop debilitating swelling attacks
NCT ID NCT05120830
First seen Apr 01, 2026 · Last updated Apr 22, 2026 · Updated 4 times
Summary
This early-stage study is testing a single-dose gene therapy called NTLA-2002 in adults with hereditary angioedema (HAE), a genetic disorder that causes sudden, severe swelling. The main goals are to see if the treatment is safe and if it can reduce the number of swelling attacks people experience. Researchers will also measure how the drug works in the body and its effect on a key protein involved in HAE attacks.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Clinical Trial Site
Campbelltown, Australia
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Clinical Trial Site
Grenoble, France
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Clinical Trial Site
Lille, France
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Clinical Trial Site
Paris, France
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Clinical Trial Site
Berlin, Germany
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Clinical Trial Site
Frankfurt, Germany
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Clinical Trial Site
Amsterdam, Netherlands
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Clinical Trial Site
Auckland, New Zealand
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Clinical Trial Site
Cambridge, United Kingdom
Conditions
Explore the condition pages connected to this study.