HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)

Last updated: June 9, 2025
Sponsor: Intellia Therapeutics
Overall Status: Active - Not Recruiting

Phase

3

Condition

Hereditary Angioedema

Allergies & Asthma

Allergy

Treatment

Normal Saline IV Administration

NTLA-2002

Clinical Study ID

NCT06634420
ITL-2002-CL-301
  • Ages > 16
  • All Genders

Study Summary

This Phase 3 study aims to evaluate the efficacy and safety of NTLA-2002 compared to placebo in participants with HAE.

Eligibility Criteria

Inclusion

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 meetthe screening requirement

  4. Must agree to refrain from the use of long-term prophylactic therapies from thestart of the screening period through the end of the Primary Observation Period. PImust 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 treatpotential angioedema attacks

  6. Adequate chemistry and hematology measures at screening

  7. Must agree not to participate in another interventional study for the duration ofthis trial.

  8. Must be capable of providing signed informed consent. Participants 16 to < 18 yearsof age, whose legal guardian provides informed consent, must provide assent.

  9. Must agree to follow contraception requirements

Exclusion

Exclusion Criteria:

  1. Concurrent diagnosis of any other type of recurrent angioedema or HAE with normalC1-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 safetyof the subject.

  4. Unwilling to comply with study procedures.

Study Design

Total Participants: 60
Treatment Group(s): 2
Primary Treatment: Normal Saline IV Administration
Phase: 3
Study Start date:
January 15, 2025
Estimated Completion Date:
September 30, 2027

Study Description

This is a multinational, multicenter, double-blind, placebo-controlled study in which approximately 60 participants will be randomized in a 2:1 ratio to receive a single IV infusion of NTLA-2002 or placebo. After the Primary Observation Period (Week 1 through Week 28), participants will have the option to receive a blinded, single IV infusion of the opposite treatment. Following the Primary Observation Period, participants will enter the Long-Term Observation Period (76 weeks), for a total of 104 weeks. Including the Screening and Run-In Period, prior to the first blinded dosing, the total study duration is approximately 28 months.

Connect with a study center

  • Campbelltown Hospital

    Campbelltown, New South Wales 2560
    Australia

    Site Not Available

  • The Alfred Hospital

    Melbourne, Victoria 3004
    Australia

    Site Not Available

  • The Alfred Hospital

    Melbourne, VIC, Victoria 3004
    Australia

    Site Not Available

  • Royal Melbourne Hospital

    Parkville, Victoria 3050
    Australia

    Site Not Available

  • University of Alberta

    Edmonton, Alberta T6G 2G3
    Canada

    Site Not Available

  • Ottawa Allergy Research Corporation

    Ottawa, Ontario K1H 1E4
    Canada

    Site Not Available

  • CHU Grenoble-Alpes - Hopital Michallon

    Grenoble, 38043
    France

    Site Not Available

  • CHU de Lille - Hopital Claude Huriez

    Lille, 59037
    France

    Site Not Available

  • AP-HM - Hopital de la Timone

    Marseille, 13385
    France

    Site Not Available

  • Charite Universitaetsmedizin Berlin

    Berlin, 12203
    Germany

    Site Not Available

  • Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet

    Frankfurt, 60590
    Germany

    Site Not Available

  • Medizinische Hochschule Hannover (MHH)

    Hannover, 30625
    Germany

    Site Not Available

  • Amsterdam UMC - Locatie AMC

    Amsterdam, 1105 AZ
    Netherlands

    Site Not Available

  • New Zealand Clinical Research (NZCR)

    Auckland, 1010
    New Zealand

    Site Not Available

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

    Cape Town, 7700
    South Africa

    Site Not Available

  • Cambridge University Hospitals NHS Foundation Trust

    Cambridge, CB2 0QQ
    United Kingdom

    Site Not Available

  • Medical Research of Arizona

    Scottsdale, Arizona 85251
    United States

    Site Not Available

  • University of California, San Diego (UCSD)

    San Diego, California 92122
    United States

    Site Not Available

  • Raffi Tachdjian MD, Inc

    Santa Monica, California 90404
    United States

    Site Not Available

  • Allergy & Asthma Clinical Research

    Walnut Creek, California 94598
    United States

    Site Not Available

  • IMMUNOe International Research Centers

    Centennial, Colorado 80112
    United States

    Site Not Available

  • Asthma & Allergy Associates

    Colorado Springs, Colorado 80907
    United States

    Site Not Available

  • University of South Florida

    Tampa, Florida 33613
    United States

    Site Not Available

  • Massachusetts General Hospital

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Henry Ford Health System

    Detriot, Michigan 48202
    United States

    Site Not Available

  • Washington University in St. Louis

    St. Louis, Missouri 63141
    United States

    Site Not Available

  • NYU Langone Health - Long Island

    Mineola, New York 11501
    United States

    Site Not Available

  • Icahn School of Medicine at Mount Sinai

    New York, New York 10029
    United States

    Site Not Available

  • Bernstein Clinical Research Center, LLC

    Cincinnati, Ohio 45236
    United States

    Site Not Available

  • Optimed Research, LTD

    Columbus, Ohio 43235
    United States

    Site Not Available

  • Penn State Milton S. Hershey Medical Center

    Hershey, Pennsylvania 17033
    United States

    Site Not Available

  • AARA Research Center

    Dallas, Texas 75231
    United States

    Site Not Available

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