HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
NCT ID: NCT06634420
Last Updated: 2025-12-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
60 participants
INTERVENTIONAL
2025-01-15
2027-09-30
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Arm A: NTLA-2002
Arm A: NTLA-2002 (50 mg; single IV infusion)
NTLA-2002
CRISPR/Cas9 gene editing system delivered by lipid nanoparticle (LNP) for intravenous (IV) administration
Arm B: Placebo
Arm B: Placebo (saline; single IV infusion)
Normal Saline IV Administration
The administration of intravenous (IV) normal saline
Interventions
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NTLA-2002
CRISPR/Cas9 gene editing system delivered by lipid nanoparticle (LNP) for intravenous (IV) administration
Normal Saline IV Administration
The administration of intravenous (IV) normal saline
Eligibility Criteria
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Inclusion Criteria
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
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.
16 Years
ALL
No
Sponsors
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Intellia Therapeutics
INDUSTRY
Responsible Party
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Locations
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Medical Research of Arizona
Scottsdale, Arizona, United States
University of California, San Diego (UCSD)
San Diego, California, United States
Raffi Tachdjian MD, Inc
Santa Monica, California, United States
Allergy & Asthma Clinical Research
Walnut Creek, California, United States
IMMUNOe International Research Centers
Centennial, Colorado, United States
Asthma & Allergy Associates
Colorado Springs, Colorado, United States
University of South Florida
Tampa, Florida, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Washington University in St. Louis
St Louis, Missouri, United States
NYU Langone Health - Long Island
Mineola, New York, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Bernstein Clinical Research Center, LLC
Cincinnati, Ohio, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
AARA Research Center
Dallas, Texas, United States
Campbelltown Hospital
Campbelltown, New South Wales, Australia
The Alfred Hospital
Melbourne, Victoria, Australia
Royal Melbourne Hospital
Parkville, Victoria, Australia
University of Alberta
Edmonton, Alberta, Canada
Ottawa Allergy Research Corporation
Ottawa, Ontario, Canada
CHU Grenoble-Alpes - Hopital Michallon
Grenoble, , France
CHU de Lille - Hopital Claude Huriez
Lille, , France
AP-HM - Hopital de la Timone
Marseille, , France
Charite Universitaetsmedizin Berlin
Berlin, , Germany
Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet
Frankfurt, , Germany
Medizinische Hochschule Hannover (MHH)
Hanover, , Germany
Amsterdam UMC - Locatie AMC
Amsterdam, , Netherlands
New Zealand Clinical Research (NZCR)
Auckland, , New Zealand
University of Cape Town - Lung Institute - Lung Clinical Research Unit
Cape Town, , South Africa
Cambridge University Hospitals NHS Foundation Trust
Cambridge, , United Kingdom
Countries
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Other Identifiers
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ITL-2002-CL-301
Identifier Type: -
Identifier Source: org_study_id