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CRISPR therapy takes on rare swelling disorder

NCT ID NCT06634420

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
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jul 30, 2026 · Updated 3 times

Summary

This phase 3 trial tests a one-time gene editing treatment called NTLA-2002 for people with hereditary angioedema (HAE), a rare genetic condition causing sudden, painful swelling attacks. About 80 participants will receive either the treatment or a placebo. The goal is to see if NTLA-2002 can reduce the number of attacks by targeting the root genetic cause.

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 using CRISPR/Cas9 delivered via lipid nanoparticles)
What this could lead to
If it works, this could provide a long-lasting reduction in HAE attacks, potentially freeing patients from regular preventive medications.
What could go wrong
This is still an early-stage trial with only 80 participants. The treatment may not work for everyone, and long-term safety is unknown. Gene editing carries risks like unintended genetic changes.

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.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

80 people

The number who actually took part.

Started

Jan 2025

Expected to finish

Sep 2027

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.

Ages

16 years and older

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. Age ≥16 years 2. Clinical history consistent with HAE-C1INH-Type 1 or -Type 2 3. Ability to provide evidence of HAE attacks (confirmed by the Investigator) to meet the screening requirement 4. Must agree to refrain from the use of long-term prophylactic therapies from the start of the screening period through the end of the Primary Observation Period. PI must be in agreement that it is medically acceptable for the participant to do so. 5. Must have access to, and the ability to use, on-demand medication(s) to treat potential angioedema attacks 6. Adequate chemistry and hematology measures at screening 7. Must agree not to participate in another interventional study for the duration of this trial. 8. Must be capable of providing signed informed consent. Participants 16 to \< 18 years of age, whose legal guardian provides informed consent, must provide assent. 9. Must agree to follow contraception requirements Exclusion Criteria: 1. Concurrent diagnosis of any other type of recurrent angioedema or HAE with normal C1-INH 2. 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.

hereditary angioedema hereditary angioedema type 1 hereditary angioedema type 2 hereditary angioedema with C1Inh deficiency

As listed by the trial registrant

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

Hereditary angioedema

Contacts and locations

Locations

  • AARA Research Center

    Dallas, Texas, 75231, United States

  • AP-HM - Hopital de la Timone

    Marseille, 13385, France

  • Allergy & Asthma Clinical Research

    Walnut Creek, California, 94598, United States

  • Amsterdam UMC - Locatie AMC

    Amsterdam, 1105 AZ, Netherlands

  • Asthma & Allergy Associates

    Colorado Springs, Colorado, 80907, United States

  • Bernstein Clinical Research Center, LLC

    Cincinnati, Ohio, 45236, United States

  • CHU Grenoble-Alpes - Hopital Michallon

    Grenoble, 38043, France

  • CHU de Lille - Hopital Claude Huriez

    Lille, 59037, France

  • Cambridge University Hospitals NHS Foundation Trust

    Cambridge, CB2 0QQ, United Kingdom

  • Campbelltown Hospital

    Campbelltown, New South Wales, 2560, Australia

  • Charite Universitaetsmedizin Berlin

    Berlin, 12203, Germany

  • IMMUNOe International Research Centers

    Centennial, Colorado, 80112, United States

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Medical Research of Arizona

    Scottsdale, Arizona, 85251, United States

  • Medizinische Hochschule Hannover (MHH)

    Hanover, 30625, Germany

  • NYU Langone Health - Long Island

    Mineola, New York, 11501, United States

  • New Zealand Clinical Research (NZCR)

    Auckland, 1010, New Zealand

  • Ottawa Allergy Research Corporation

    Ottawa, Ontario, K1H 1E4, Canada

  • Penn State Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033, United States

  • Raffi Tachdjian MD, Inc

    Santa Monica, California, 90404, United States

  • Royal Melbourne Hospital

    Parkville, Victoria, 3050, Australia

  • The Alfred Hospital

    Melbourne, Victoria, 3004, Australia

  • Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet

    Frankfurt, 60590, Germany

  • University of Alberta

    Edmonton, Alberta, T6G 2G3, Canada

  • University of California, San Diego (UCSD)

    San Diego, California, 92122, United States

  • University of Cape Town - Lung Institute - Lung Clinical Research Unit

    Cape Town, 7700, South Africa

  • University of South Florida

    Tampa, Florida, 33613, United States

  • Washington University in St. Louis

    St Louis, Missouri, 63141, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.