Viewing Study NCT06563895


Ignite Creation Date: 2025-12-24 @ 1:42 PM
Ignite Modification Date: 2026-03-22 @ 10:44 AM
Study NCT ID: NCT06563895
Status: RECRUITING
Last Update Posted: 2025-12-23
First Post: 2024-08-05
Is NOT Gene Therapy: True
Has Adverse Events: False

Brief Title: Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
Sponsor:
Organization:

Raw JSON

{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Bulgaria']}, 'conditionBrowseModule': {'meshes': [{'id': 'D000686', 'term': 'Amyloidosis'}, {'id': 'D028227', 'term': 'Amyloid Neuropathies, Familial'}, {'id': 'C567782', 'term': 'Amyloidosis, Hereditary, Transthyretin-Related'}, {'id': 'D009202', 'term': 'Cardiomyopathies'}, {'id': 'D006331', 'term': 'Heart Diseases'}, {'id': 'D011115', 'term': 'Polyneuropathies'}, {'id': 'D000544', 'term': 'Alzheimer Disease'}], 'ancestors': [{'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D017772', 'term': 'Amyloid Neuropathies'}, {'id': 'D010523', 'term': 'Peripheral Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D028226', 'term': 'Amyloidosis, Familial'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D003704', 'term': 'Dementia'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D024801', 'term': 'Tauopathies'}, {'id': 'D019965', 'term': 'Neurocognitive Disorders'}, {'id': 'D001523', 'term': 'Mental Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000731204', 'term': 'attruby'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'PREVENTION', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 582}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2025-05-12', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2032-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-19', 'studyFirstSubmitDate': '2024-08-05', 'studyFirstSubmitQcDate': '2024-08-18', 'lastUpdatePostDateStruct': {'date': '2025-12-23', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-08-21', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2031-10', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Time to development of ATTR (ATTR-CM or ATTR-PN, whichever occurs first; centrally adjudicated)', 'timeFrame': 'Since randomization up to approximately 7 years or until the study is declared over', 'description': '* ATTR-CM defined by biopsy or imaging-based diagnosis\n* ATTR-PN defined by new signs or symptoms and biopsy-based diagnosis'}], 'secondaryOutcomes': [{'measure': 'Time to development of ATTR-CM (centrally adjudicated)', 'timeFrame': 'Since randomization up to approximately 7 years or until the study is declared over', 'description': 'ATTR-CM defined by biopsy or imaging-based diagnosis'}, {'measure': 'Time to development of ATTR-PN (centrally adjudicated)', 'timeFrame': 'Since randomization up to approximately 7 years or until the study is declared over', 'description': 'ATTR-PN defined by new signs or symptoms and biopsy-based diagnosis'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Amyloidosis', 'ATTR-CM', 'ATTR-PN', 'Transthyretin', 'Amyloid', 'TTR'], 'conditions': ['Amyloidosis', 'Amyloid Cardiomyopathy', 'Transthyretin Amyloidosis', 'Cardiomyopathies', 'Heart Diseases', 'Polyneuropathies']}, 'descriptionModule': {'briefSummary': 'Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque- like substance that accumulates in different organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall apart. One way occurs as a person ages, where the normal TTR protein can fall apart and form amyloid that may no longer be sufficiently cleared by the body. This type of ATTR is known as wild-type ATTR (ATTRwt). The other way occurs when a person inherits a defective TTR gene that causes the TTR protein to spontaneously fall apart. This form of the disease is known as variant ATTR (ATTRv) and can be detected in adults by a genetic test of their TTR gene before they age.\n\nAmyloid build-up in the heart causes the heart wall to become thick and stiff and can result in heart failure and even death. Accumulation of TTR amyloid in the heart is known as transthyretin amyloid cardiomyopathy or ATTR-CM. Amyloid can also deposit in the nerve tissues leading to nerve problems. Accumulation of TTR in the nerves is known as transthyretin amyloid polyneuropathy or ATTR-PN.\n\nAcoramidis is an experimental drug designed to bind tightly to TTR in the blood and stabilize its structure, so it does not form the harmful amyloid plaques that can cause damage to organs.\n\nThis study is intended to determine if treatment with acoramidis in participants with ATTRv who have not yet developed any symptoms of disease can prevent or delay the development of ATTR-CM or ATTR-PN disease. If adults with an inherited defective TTR gene are treated early before any of the symptoms of disease have developed, it may be possible to delay the onset or prevent the disease entirely.', 'detailedDescription': 'The AG10-501 ACT-EARLY study is a randomized, multicenter, double-blind, placebo- controlled study of acoramidis for prevention of ATTR (with specific reference to either its cardiomyopathic or polyneuropathic manifestations). Participants will be stratified at randomization.\n\nThe study population will be asymptomatic carriers of a known pathogenic TTR gene variant. A participant must be 18 to 75 inclusive years of age, and the age of the participant must be no more than 10 years younger than the predicted age of disease onset (PADO) based either on family history (pedigree analysis) or, if family history is insufficient, based on a TTR Variant Actuarial table from published literature. For example, if PADO for a given individual is found to be 50 years, the age of the participant must be between 40 and 75 years inclusive.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Key Inclusion Criteria:\n\n* Male or female ≥ 18 to ≤ 75 years of age inclusive.\n* Participants must have an established genotype (hetero- or homozygosity) of a TTR gene variant that is known to be pathogenic (eg, V30M/p.V50M, V122I/p.V142I, T60A/p.T80A, or any other pathogenic TTR variant(s)) confirmed by central laboratory prior to randomization.\n* Participant's age is no more than 10 years (≤ 10) younger than the PADO.\n\nKey Exclusion Criteria:\n\n* Evidence of ATTR-CM or ATTR-PN.\n* Presence of a TTR variant known to be phenotypically protective (eg, T119M, R104H).\n* Current or past treatment with other TTR modifying therapies.\n* Contraindication to or inability to undergo Cardiac magnetic resonance testing.\n* Major organ dysfunction, including: kidney disease, liver disease, heart disease (including cardiomyopathy), neuropathy\n* Other diseases or conditions such has cancer within 3 years, untreated hyperthyroidism or hypothyroidism, type 1 diabetes, active hepatitis B or C, HIV.\n* Major surgery within the past 3 months or planned during the next 12 months.\n* Known hypersensitivity to acoramidis."}, 'identificationModule': {'nctId': 'NCT06563895', 'briefTitle': 'Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant', 'organization': {'class': 'INDUSTRY', 'fullName': 'Eidos Therapeutics, a BridgeBio company'}, 'officialTitle': 'A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)', 'orgStudyIdInfo': {'id': 'AG10-501'}, 'secondaryIdInfos': [{'id': 'U1111-1307-8107', 'type': 'OTHER', 'domain': 'WHO'}, {'id': 'jRCT2071250116', 'type': 'OTHER', 'domain': 'Japan Registry of Clinical Trials'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'acoramidis', 'description': 'Participants will receive acoramidis 712 mg orally BID (which is equivalent to 800 mg acoramidis HCl BID)', 'interventionNames': ['Drug: Acoramidis']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Subjects will receive placebo to match twice daily', 'interventionNames': ['Drug: Placebo oral tablet']}], 'interventions': [{'name': 'Acoramidis', 'type': 'DRUG', 'otherNames': ['AG10', 'ALXN2060'], 'description': 'TTR stabilizer administered orally twice daily (BID)', 'armGroupLabels': ['acoramidis']}, {'name': 'Placebo oral tablet', 'type': 'DRUG', 'description': 'Non-active control administered orally twice daily (BID)', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92037', 'city': 'La Jolla', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'University of California, San Diego (UCSD) - Medical Center', 'geoPoint': {'lat': 32.84727, 'lon': -117.2742}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'University of California, Los Angeles (UCLA) - David Geffen School of Medicine', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94143', 'city': 'San Francisco', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'University of California, San Francisco (UCSF)', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '94305', 'city': 'Stanford', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'University of Colorado Anschutz', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '06519', 'city': 'New Haven', 'state': 'Connecticut', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Yale University School of Medicine - Section of Cardiology', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}, {'zip': '20010', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'MedStar Washington Hospital Center - MedStar Heart and Vascular Institute', 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '32224', 'city': 'Jacksonville', 'state': 'Florida', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Mayo Clinic - Jacksonville', 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Emory University School of Medicine', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'John H. 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