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

July 28, 2026 updated by: Intellia Therapeutics

HAELO: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2002 in Participants With Hereditary Angioedema (HAE)

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed 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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
80

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Australia
    • New South Wales
      • Campbelltown, New South Wales, Australia, 2560
        • Campbelltown Hospital
    • Victoria
      • Melbourne, Victoria, Australia, 3004
        • The Alfred Hospital
      • Parkville, Victoria, Australia, 3050
        • Royal Melbourne Hospital
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2G3
        • University of Alberta
    • Ontario
      • Ottawa, Ontario, Canada, K1H 1E4
        • Ottawa Allergy Research Corporation
  • France
      • Grenoble, France, 38043
        • CHU Grenoble-Alpes - Hopital Michallon
      • Lille, France, 59037
        • CHU de Lille - Hôpital Claude Huriez
      • Marseille, France, 13385
        • AP-HM - Hopital de la Timone
  • Germany
      • Berlin, Germany, 12203
        • Charite Universitaetsmedizin Berlin
      • Frankfurt, Germany, 60590
        • Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet
      • Hanover, Germany, 30625
        • Medizinische Hochschule Hannover (MHH)
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Amsterdam UMC - Locatie AMC
  • New Zealand
      • Auckland, New Zealand, 1010
        • New Zealand Clinical Research (NZCR)
  • South Africa
      • Cape Town, South Africa, 7700
        • University of Cape Town - Lung Institute - Lung Clinical Research Unit
  • United Kingdom
      • Cambridge, United Kingdom, CB2 0QQ
        • Cambridge University Hospitals NHS Foundation Trust
  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85251
        • Medical Research of Arizona
    • California
      • San Diego, California, United States, 92122
        • University of California, San Diego (UCSD)
      • Santa Monica, California, United States, 90404
        • Raffi Tachdjian MD, Inc
      • Walnut Creek, California, United States, 94598
        • Allergy & Asthma Clinical Research
    • Colorado
      • Centennial, Colorado, United States, 80112
        • IMMUNOe International Research Centers
      • Colorado Springs, Colorado, United States, 80907
        • Asthma & Allergy Associates
    • Florida
      • Tampa, Florida, United States, 33613
        • University of South Florida
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
    • Missouri
      • St Louis, Missouri, United States, 63141
        • Washington University in St. Louis
    • New York
      • Mineola, New York, United States, 11501
        • NYU Langone Health - Long Island
      • New York, New York, United States, 10029
        • Icahn School of Medicine at Mount Sinai
    • Ohio
      • Cincinnati, Ohio, United States, 45236
        • Bernstein Clinical Research Center, LLC
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Penn State Milton S. Hershey Medical Center
    • Texas
      • Dallas, Texas, United States, 75231
        • AARA Research Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Active Comparator: Arm A: NTLA-2002
Arm A: NTLA-2002 (50 mg; single IV infusion)
Biological: NTLA-2002
CRISPR/Cas9 gene editing system delivered by lipid nanoparticle (LNP) for intravenous (IV) administration
Placebo Comparator: Arm B: Placebo
Arm B: Placebo (saline; single IV infusion)
Biological: Normal Saline IV Administration
The administration of intravenous (IV) normal saline

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Time-normalized number of Investigator-confirmed HAE attacks
Time Frame: From Week 5 through Week 28
From Week 5 through Week 28

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Time-normalized number of Investigator-confirmed HAE attacks
Time Frame: From Week 1 through Week 28
From Week 1 through Week 28
Time-normalized number of Investigator-confirmed HAE attacks requiring on-demand treatment
Time Frame: From Week 5 through Week 28
From Week 5 through Week 28
Time-normalized number of moderate or severe Investigator-confirmed HAE attacks
Time Frame: From Week 5 through Week 28
From Week 5 through Week 28
Investigator-confirmed HAE attack-free status
Time Frame: From Week 5 through Week 28
From Week 5 through Week 28
Change from baseline to Week 28 in AE-QoL Questionnaire total score
Time Frame: From baseline to Week 28
From baseline to Week 28
Responder status, defined as reduction from baseline of at least 50%, 70%, and 90% in time-normalized number of Investigator-confirmed HAE attacks
Time Frame: From Week 5 through Week 28
From Week 5 through Week 28
Time-normalized number of Investigator-confirmed HAE attacks requiring on-demand treatment
Time Frame: from Week 1 through Week 28
from Week 1 through Week 28
Time-normalized number of moderate or severe Investigator-confirmed HAE attacks
Time Frame: From Week 1 through Week 28
From Week 1 through Week 28
Investigator-confirmed HAE attack-free status
Time Frame: From Week 1 through Week 28
From Week 1 through Week 28
Reduction from baseline of at least 50%, 70%, and 90% in time-normalized number of Investigator-confirmed HAE attacks
Time Frame: From Week 1 through Week 28
From Week 1 through Week 28
Long-term prophylaxis-free status
Time Frame: From Week 5 through Week 104
From Week 5 through Week 104
HAE treatment-free status, defined as no usage of long-term prophylaxis or on-demand medication
Time Frame: From Week 5 through Week 104
From Week 5 through Week 104
Complete responder status, defined as zero Investigator-confirmed attacks with no long-term prophylaxis
Time Frame: From Week 5 through Week 104
From Week 5 through Week 104
Reduction in the time-normalized number of Investigator-confirmed HAE attacks after crossover treatment (Week 33 through Week 104) compared to before crossover treatment (Screening through Week 28) in participants randomized to placebo
Time Frame: From Week 33 through Week 104 and from Screening through Week 28
From Week 33 through Week 104 and from Screening through Week 28

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Intellia Therapeutics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 15, 2025

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 10, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 7, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 8, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 9, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 29, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 28, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ITL-2002-CL-301

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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