Gene therapy for HAE: does it last? new study tracks Long-Term safety
NCT ID NCT06262399
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This study follows about 100 people with hereditary angioedema who previously received the gene-editing therapy NTLA-2002. Researchers will monitor side effects and how well the treatment controls swelling attacks over time. The goal is to understand if a single dose can provide lasting relief.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- NTLA-2002 (a gene-editing therapy)
- What this could lead to
- If successful, this could show that a single dose of NTLA-2002 safely reduces or prevents HAE attacks over many years, pointing toward a long-term treatment.
- What could go wrong
- This is an observational follow-up, not a new treatment test. It may reveal side effects or waning effectiveness over time. The therapy is still experimental.
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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Cambridge University Hospitals NHS Foundation Trust, Addenbrooke's Hospital
Cambridge, United Kingdom
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Campbelltown Hospital
Campbelltown, New South Wales, 2560, Australia
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Centre National de Reference - Grenoble
Grenoble, France
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Charite-Universitätsmedizin Berlin
Berlin, 12203, Germany
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Hôpital Claude Huriez
Lille, 59037, France
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New Zealand Clinical Research
Auckland, New Zealand
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University of Amsterdam Academic Medical Center
Amsterdam, Netherlands
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New oral option may offer relief for hereditary angioedema attacks
- Can a new injection tame hereditary swelling attacks?
- Could a simple pill shield HAE patients from Procedure-Triggered attacks?
- Teens with rare swelling disorder get new drug tested
- New drug shows promise for controlling rare swelling disorder
- New drug navenibart aims to tame hereditary swelling attacks