Gene therapy aims to stop painful swelling attacks in hereditary angioedema
NCT ID NCT05120830
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new treatment called NTLA-2002 for adults with hereditary angioedema (HAE), a condition that causes sudden, painful swelling attacks. The therapy uses gene editing to lower a protein that triggers these attacks. The goal is to see if it is safe and can reduce how often attacks happen.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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37 people
The number who actually took part.
- Started
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Dec 2021
- Expected to finish
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Jul 2026
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Age \>18 years 2. Diagnosis of HAE Types I or II 3. Ability to provide evidence of HAE attacks to meet the screening requirement 4. Subjects must have access to, and the ability to use, ≥ 1 acute medication(s) to treat angioedema attacks. 5. Adequate chemistry and hematology measures at screening 6. Subjects must agree not to participate in another interventional study for the duration of this trial. 7. Subjects must be capable of providing signed informed consent Exclusion Criteria: 1. Concurrent diagnosis of any other type of recurrent angioedema 2. Subjects who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component. 3. Any condition that, in the Investigator's opinion, could adversely affect the safety of the subject. 4. Unwilling to comply with study procedures.
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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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Clinical Trial Site
Campbelltown, Australia
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Clinical Trial Site
Grenoble, France
-
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Could a daily pill stop hereditary angioedema attacks?
- 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