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

NCT06634420 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: ITL-2002-CL-301
• Study Type: interventional
• Target: 60
• Locations: 9 countries
• Sites: 29

Brief Summary

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

Conditions

Hereditary Angioedema

Keywords

Hereditary Angioedema; NTLA-2002; HAE; HAE-C1INH-Type1 or -Type 2

Outcome Measures

Primary Outcomes (1)

• Time-normalized number of Investigator-confirmed HAE attacks

  From Week 5 through Week 28

Secondary Outcomes (14)

• Time-normalized number of Investigator-confirmed HAE attacks requiring on-demand treatment

  From Week 5 through Week 28

• Time-normalized number of moderate or severe Investigator-confirmed HAE attacks

  From Week 5 through Week 28

• Investigator-confirmed HAE attack-free status

  From Week 5 through Week 28

• Change from baseline to Week 28 in AE-QoL Questionnaire total score

  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

  From Week 5 through Week 28

Study Arms (2)

Arm A: NTLA-2002

ACTIVE COMPARATOR

Arm A: NTLA-2002 (50 mg; single IV infusion)

Biological: NTLA-2002

Arm B: Placebo

PLACEBO COMPARATOR

Arm B: Placebo (saline; single IV infusion)

Biological: Normal Saline IV Administration

Interventions

NTLA-2002 BIOLOGICAL

CRISPR/Cas9 gene editing system delivered by lipid nanoparticle (LNP) for intravenous (IV) administration

Arm A: NTLA-2002

Normal Saline IV Administration BIOLOGICAL

The administration of intravenous (IV) normal saline

Arm B: Placebo

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥16 years
• Clinical history consistent with HAE-C1INH-Type 1 or -Type 2
• Ability to provide evidence of HAE attacks (confirmed by the Investigator) to meet the screening requirement
• 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.
• Must have access to, and the ability to use, on-demand medication(s) to treat potential angioedema attacks
• Adequate chemistry and hematology measures at screening
• Must agree not to participate in another interventional study for the duration of this trial.
• Must be capable of providing signed informed consent. Participants 16 to \< 18 years of age, whose legal guardian provides informed consent, must provide assent.
• Must agree to follow contraception requirements

You may not qualify if:

• Concurrent diagnosis of any other type of recurrent angioedema or HAE with normal C1-INH
• Have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
• Any condition that, in the Investigator's opinion, could adversely affect the safety of the subject.
• Unwilling to comply with study procedures.

Sponsors & Collaborators

• Intellia Therapeutics: lead

Study Sites (29)

Medical Research of Arizona

Scottsdale, Arizona, 85251, United States

Location

University of California, San Diego (UCSD)

San Diego, California, 92122, United States

Location

Raffi Tachdjian MD, Inc

Santa Monica, California, 90404, United States

Location

Allergy & Asthma Clinical Research

Walnut Creek, California, 94598, United States

Location

IMMUNOe International Research Centers

Centennial, Colorado, 80112, United States

Location

Asthma & Allergy Associates

Colorado Springs, Colorado, 80907, United States

Location

University of South Florida

Tampa, Florida, 33613, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Washington University in St. Louis

St Louis, Missouri, 63141, United States

Location

NYU Langone Health - Long Island

Mineola, New York, 11501, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

Bernstein Clinical Research Center, LLC

Cincinnati, Ohio, 45236, United States

Location

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

AARA Research Center

Dallas, Texas, 75231, United States

Location

Campbelltown Hospital

Campbelltown, New South Wales, 2560, Australia

Location

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

Location

Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia

Location

University of Alberta

Edmonton, Alberta, T6G 2G3, Canada

Location

Ottawa Allergy Research Corporation

Ottawa, Ontario, K1H 1E4, Canada

Location

CHU Grenoble-Alpes - Hopital Michallon

Grenoble, 38043, France

Location

CHU de Lille - Hopital Claude Huriez

Lille, 59037, France

Location

AP-HM - Hopital de la Timone

Marseille, 13385, France

Location

Charite Universitaetsmedizin Berlin

Berlin, 12203, Germany

Location

Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet

Frankfurt, 60590, Germany

Location

Medizinische Hochschule Hannover (MHH)

Hanover, 30625, Germany

Location

Amsterdam UMC - Locatie AMC

Amsterdam, 1105 AZ, Netherlands

Location

New Zealand Clinical Research (NZCR)

Auckland, 1010, New Zealand

Location

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

Cape Town, 7700, South Africa

Location

Cambridge University Hospitals NHS Foundation Trust

Cambridge, CB2 0QQ, United Kingdom

Location

MeSH Terms

Conditions

Angioedemas, Hereditary

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 7, 2024
• First Posted: October 9, 2024
• Study Start: January 15, 2025
• Primary Completion: April 1, 2026
• Study Completion (Estimated): September 1, 2027
• Last Updated: December 18, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

NL (1); FR (3); CA (2); DE (3); AU (3); GB (1); US (14); NZ (1); ZA (1)