Ignite Creation Date:
2025-12-25 @ 4:24 AM
Ignite Modification Date:
2026-01-10 @ 4:16 AM
Study NCT ID:
NCT06634420
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-12-18
First Post:
2024-10-07
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D054179', 'term': 'Angioedemas, Hereditary'}], 'ancestors': [{'id': 'D000799', 'term': 'Angioedema'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D000081208', 'term': 'Hereditary Complement Deficiency Diseases'}, {'id': 'D000081207', 'term': 'Primary Immunodeficiency Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D014581', 'term': 'Urticaria'}, {'id': 'D017445', 'term': 'Skin Diseases, Vascular'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D006969', 'term': 'Hypersensitivity, Immediate'}, {'id': 'D006967', 'term': 'Hypersensitivity'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 60}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2025-01-15', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-06', 'completionDateStruct': {'date': '2027-09', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-17', 'studyFirstSubmitDate': '2024-10-07', 'studyFirstSubmitQcDate': '2024-10-08', 'lastUpdatePostDateStruct': {'date': '2025-12-18', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-10-09', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-04', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Time-normalized number of Investigator-confirmed HAE attacks', 'timeFrame': 'From Week 5 through Week 28'}], 'secondaryOutcomes': [{'measure': 'Time-normalized number of Investigator-confirmed HAE attacks requiring on-demand treatment', 'timeFrame': 'From Week 5 through Week 28'}, {'measure': 'Time-normalized number of moderate or severe Investigator-confirmed HAE attacks', 'timeFrame': 'From Week 5 through Week 28'}, {'measure': 'Investigator-confirmed HAE attack-free status', 'timeFrame': 'From Week 5 through Week 28'}, {'measure': 'Change from baseline to Week 28 in AE-QoL Questionnaire total score', 'timeFrame': 'From baseline to Week 28'}, {'measure': 'Responder status, defined as reduction from baseline of at least 50%, 70%, and 90% in time-normalized number of Investigator-confirmed HAE attacks', 'timeFrame': 'From Week 5 through Week 28'}, {'measure': 'Time-normalized number of Investigator-confirmed HAE attacks', 'timeFrame': 'From Week 1 through Week 28'}, {'measure': 'Time-normalized number of Investigator-confirmed HAE attacks requiring on-demand treatment', 'timeFrame': 'from Week 1 through Week 28'}, {'measure': 'Time-normalized number of moderate or severe Investigator-confirmed HAE attacks', 'timeFrame': 'From Week 1 through Week 28'}, {'measure': 'Investigator-confirmed HAE attack-free status', 'timeFrame': 'From Week 1 through Week 28'}, {'measure': 'Reduction from baseline of at least 50%, 70%, and 90% in time-normalized number of Investigator-confirmed HAE attacks', 'timeFrame': 'From Week 1 through Week 28'}, {'measure': 'Long-term prophylaxis-free status', 'timeFrame': 'From Week 5 through Week 104'}, {'measure': 'HAE treatment-free status, defined as no usage of long-term prophylaxis or on-demand medication', 'timeFrame': 'From Week 5 through Week 104'}, {'measure': 'Complete responder status, defined as zero Investigator-confirmed attacks with no long-term prophylaxis', 'timeFrame': 'From Week 5 through Week 104'}, {'measure': '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', 'timeFrame': 'From Week 33 through Week 104 and from Screening through Week 28'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Hereditary Angioedema', 'NTLA-2002', 'HAE', 'HAE-C1INH-Type1 or -Type 2'], 'conditions': ['Hereditary Angioedema']}, 'descriptionModule': {'briefSummary': 'This Phase 3 study aims to evaluate the efficacy and safety of NTLA-2002 compared to placebo in participants with HAE.', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Age ≥16 years\n2. Clinical history consistent with HAE-C1INH-Type 1 or -Type 2\n3. Ability to provide evidence of HAE attacks (confirmed by the Investigator) to meet the screening requirement\n4. 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.\n5. Must have access to, and the ability to use, on-demand medication(s) to treat potential angioedema attacks\n6. Adequate chemistry and hematology measures at screening\n7. Must agree not to participate in another interventional study for the duration of this trial.\n8. Must be capable of providing signed informed consent. Participants 16 to \\< 18 years of age, whose legal guardian provides informed consent, must provide assent.\n9. Must agree to follow contraception requirements\n\nExclusion Criteria:\n\n1. Concurrent diagnosis of any other type of recurrent angioedema or HAE with normal C1-INH\n2. Have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.\n3. Any condition that, in the Investigator's opinion, could adversely affect the safety of the subject.\n4. Unwilling to comply with study procedures."}, 'identificationModule': {'nctId': 'NCT06634420', 'briefTitle': 'HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Intellia Therapeutics'}, 'officialTitle': '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)', 'orgStudyIdInfo': {'id': 'ITL-2002-CL-301'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'Arm A: NTLA-2002', 'description': 'Arm A: NTLA-2002 (50 mg; single IV infusion)', 'interventionNames': ['Biological: NTLA-2002']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Arm B: Placebo', 'description': 'Arm B: Placebo (saline; single IV infusion)', 'interventionNames': ['Biological: Normal Saline IV Administration']}], 'interventions': [{'name': 'NTLA-2002', 'type': 'BIOLOGICAL', 'description': 'CRISPR/Cas9 gene editing system delivered by lipid nanoparticle (LNP) for intravenous (IV) administration', 'armGroupLabels': ['Arm A: NTLA-2002']}, {'name': 'Normal Saline IV Administration', 'type': 'BIOLOGICAL', 'description': 'The administration of intravenous (IV) normal saline', 'armGroupLabels': ['Arm B: Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85251', 'city': 'Scottsdale', 'state': 'Arizona', 'country': 'United States', 'facility': 'Medical Research of Arizona', 'geoPoint': {'lat': 33.50921, 'lon': -111.89903}}, {'zip': '92122', 'city': 'San Diego', 'state': 'California', 'country': 'United States', 'facility': 'University of California, San Diego (UCSD)', 'geoPoint': {'lat': 32.71571, 'lon': -117.16472}}, {'zip': '90404', 'city': 'Santa Monica', 'state': 'California', 'country': 'United States', 'facility': 'Raffi Tachdjian MD, Inc', 'geoPoint': {'lat': 34.01949, 'lon': -118.49138}}, {'zip': '94598', 'city': 'Walnut Creek', 'state': 'California', 'country': 'United States', 'facility': 'Allergy & Asthma Clinical Research', 'geoPoint': {'lat': 37.90631, 'lon': -122.06496}}, {'zip': '80112', 'city': 'Centennial', 'state': 'Colorado', 'country': 'United States', 'facility': 'IMMUNOe International Research Centers', 'geoPoint': {'lat': 39.57916, 'lon': -104.87692}}, {'zip': '80907', 'city': 'Colorado Springs', 'state': 'Colorado', 'country': 'United States', 'facility': 'Asthma & Allergy Associates', 'geoPoint': {'lat': 38.83388, 'lon': -104.82136}}, {'zip': '33613', 'city': 'Tampa', 'state': 'Florida', 'country': 'United States', 'facility': 'University of South Florida', 'geoPoint': {'lat': 27.94752, 'lon': -82.45843}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '63141', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University in St. Louis', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '11501', 'city': 'Mineola', 'state': 'New York', 'country': 'United States', 'facility': 'NYU Langone Health - Long Island', 'geoPoint': {'lat': 40.74927, 'lon': -73.64068}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Icahn School of Medicine at Mount Sinai', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '45236', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': 'Bernstein Clinical Research Center, LLC', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '17033', 'city': 'Hershey', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Penn State Milton S. Hershey Medical Center', 'geoPoint': {'lat': 40.28592, 'lon': -76.65025}}, {'zip': '75231', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'AARA Research Center', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '2560', 'city': 'Campbelltown', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'Campbelltown Hospital', 'geoPoint': {'lat': -34.06667, 'lon': 150.81667}}, {'zip': '3004', 'city': 'Melbourne', 'state': 'Victoria', 'country': 'Australia', 'facility': 'The Alfred Hospital', 'geoPoint': {'lat': -37.814, 'lon': 144.96332}}, {'zip': '3050', 'city': 'Parkville', 'state': 'Victoria', 'country': 'Australia', 'facility': 'Royal Melbourne Hospital', 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'zip': 'T6G 2G3', 'city': 'Edmonton', 'state': 'Alberta', 'country': 'Canada', 'facility': 'University of Alberta', 'geoPoint': {'lat': 53.55014, 'lon': -113.46871}}, {'zip': 'K1H 1E4', 'city': 'Ottawa', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Ottawa Allergy Research Corporation', 'geoPoint': {'lat': 45.41117, 'lon': -75.69812}}, {'zip': '38043', 'city': 'Grenoble', 'country': 'France', 'facility': 'CHU Grenoble-Alpes - Hopital Michallon', 'geoPoint': {'lat': 45.17869, 'lon': 5.71479}}, {'zip': '59037', 'city': 'Lille', 'country': 'France', 'facility': 'CHU de Lille - Hopital Claude Huriez', 'geoPoint': {'lat': 50.63391, 'lon': 3.05512}}, {'zip': '13385', 'city': 'Marseille', 'country': 'France', 'facility': 'AP-HM - Hopital de la Timone', 'geoPoint': {'lat': 43.29695, 'lon': 5.38107}}, {'zip': '12203', 'city': 'Berlin', 'country': 'Germany', 'facility': 'Charite Universitaetsmedizin Berlin', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'zip': '60590', 'city': 'Frankfurt', 'country': 'Germany', 'facility': 'Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet', 'geoPoint': {'lat': 49.68333, 'lon': 10.53333}}, {'zip': '30625', 'city': 'Hanover', 'country': 'Germany', 'facility': 'Medizinische Hochschule Hannover (MHH)', 'geoPoint': {'lat': 52.37052, 'lon': 9.73322}}, {'zip': '1105 AZ', 'city': 'Amsterdam', 'country': 'Netherlands', 'facility': 'Amsterdam UMC - Locatie AMC', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}, {'zip': '1010', 'city': 'Auckland', 'country': 'New Zealand', 'facility': 'New Zealand Clinical Research (NZCR)', 'geoPoint': {'lat': -36.84853, 'lon': 174.76349}}, {'zip': '7700', 'city': 'Cape Town', 'country': 'South Africa', 'facility': 'University of Cape Town - Lung Institute - Lung Clinical Research Unit', 'geoPoint': {'lat': -33.92584, 'lon': 18.42322}}, {'zip': 'CB2 0QQ', 'city': 'Cambridge', 'country': 'United Kingdom', 'facility': 'Cambridge University Hospitals NHS Foundation Trust', 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Intellia Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}