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Gene therapy for HAE: does it last? new study tracks Long-Term safety

NCT ID NCT06262399

Disease control Sponsor: Intellia Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 100 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2024

Expected to finish

Apr 2040

An estimate. End dates often move.

Lead sponsor

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.

Who is studied

Participating sites in an Intellia-sponsored clinical study of NTLA-2002.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. A subject has completed or discontinued from an Intellia-sponsored or -supported treatment protocol in which a complete or partial dose of NTLA-2002 was received. 2. A subject has provided informed consent for the LTFU study. 3. A subject is willing to attend study visits, complete protocol-required follow-up schedule, and comply with the study requirements. Exclusion Criteria: None

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Conditions

The condition(s) this trial relates to.

hereditary angioedema

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hereditary angioedema

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    7 sites in 6 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Cambridge University Hospitals NHS Foundation Trust, Addenbrooke's Hospital

    Cambridge, United Kingdom

  • Campbelltown Hospital

    Campbelltown, New South Wales, 2560, Australia

  • Centre National de Reference - Grenoble

    Grenoble, France

  • Charite-Universitätsmedizin Berlin

    Berlin, 12203, Germany

  • Hôpital Claude Huriez

    Lille, 59037, France

  • New Zealand Clinical Research

    Auckland, New Zealand

  • University of Amsterdam Academic Medical Center

    Amsterdam, Netherlands

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