Ignite Creation Date:
2025-12-24 @ 6:37 PM
Ignite Modification Date:
2026-01-04 @ 8:33 AM
Study NCT ID:
NCT05797857
Status:
RECRUITING
Last Update Posted:
2025-03-18
First Post:
2023-03-22
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Exercise Training in Transthyretin Cardiac Amyloidosis
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D028227', 'term': 'Amyloid Neuropathies, Familial'}], 'ancestors': [{'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': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D000686', 'term': 'Amyloidosis'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D015444', 'term': 'Exercise'}], 'ancestors': [{'id': 'D009043', 'term': 'Motor Activity'}, {'id': 'D009068', 'term': 'Movement'}, {'id': 'D009142', 'term': 'Musculoskeletal Physiological Phenomena'}, {'id': 'D055687', 'term': 'Musculoskeletal and Neural Physiological Phenomena'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 40}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-07-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-03', 'completionDateStruct': {'date': '2030-06-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-03-14', 'studyFirstSubmitDate': '2023-03-22', 'studyFirstSubmitQcDate': '2023-03-22', 'lastUpdatePostDateStruct': {'date': '2025-03-18', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-04-04', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2029-06-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'peak oxygen consumption (VO2)', 'timeFrame': '12 weeks', 'description': 'CPET performed at baseline and 12-weeks, following the exercise intervention will be used to measure aerobic capacity, peak VO2. The change in peak VO2 from baseline to 12 weeks is the primary outcome measure. An increase of \\> 1.0 ml/kg/min is considered a clinically meaningful increase'}], 'secondaryOutcomes': [{'measure': 'Kansas City Cardiomyopathy Questionnaire', 'timeFrame': '12 weeks', 'description': 'Kansas City Cardiomyopathy Questionnaire (KCCQ) is a quality of life questionnaire. This will be recorded at baseline, 4-weeks and 12-weeks. The change in KCCQ score from baseline to 12 weeks is a secondary outcome. KCCQ scores are scaled from 0 to 100; where 0 denotes the lowest reportable health status and 100 the highest. An increase of 5 points is considered a clinically meaningful increase.'}, {'measure': 'Lower extremity function', 'timeFrame': '12 weeks', 'description': 'The short physical performance battery (SPPB) is an assessment of lower extremity function. The change in SPPB score from baseline to 12 weeks is a secondary outcome. This is a scale of 0-12, where 0 denotes the worst performance and 12 the highest. An increase of 1 point is considered a clinically meaningful increase.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Amyloid Cardiomyopathy', 'Transthyretin Cardiac Amyloidosis']}, 'descriptionModule': {'briefSummary': 'Transthyretin cardiac amyloidosis causes debilitating heart failure in older adults. The proposed research will develop a personalized exercise training program to improve functional capacity in patients on optimal treatment for transthyretin cardiac amyloidosis. This is a vital next step to improve functional capacity and quality of life of people suffering from transthyretin cardiac amyloidosis.', 'detailedDescription': 'Heart failure (HF) affects over 5 million adults over the age of 65. Cardiac transthyretin amyloidosis (ATTR-CM) is a cause of HF in \\~10% of older adults and leads to significant morbidity and mortality. Exercise intolerance is traditionally attributed to cardiac dysfunction but the contribution of other systems to this has not been studied. Musculoskeletal involvement is common in ATTR-CM and occur 5-10 years prior to onset of HF. Tafamidis, a transthyretin stabilizer, is the only approved treatment for ATTR-CM. It slows disease progression, prolongs life, and reduces HF hospitalizations. However, it does not improve functional capacity- no therapeutic intervention has been shown to do so in ATTR-CM.\n\nThe idea behind this project is that skeletal muscle dysfunction from amyloidosis and HF severely limits exercise capacity and, thus, quality of life in ATTR-CM, and that targeted exercise training will improve quality of life by improving skeletal muscle performance and aerobic capacity. Cardiopulmonary exercise testing (CPET) and the short physical performance battery (SBBP), including a leg extensor muscle power assessment will be used to achieve the following specific aims; 1) to compare skeletal muscle performance in ATTR-CM and non-amyloid HF; and 2) to determine improvements in aerobic capacity and quality of life due to 12 weeks of supervised exercise training in patients with ATTR-CM. To achieve the second aim, we will use a personalized exercise intervention.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '90 Years', 'minimumAge': '60 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis and typing of ATTR-CM by endomyocardial biopsy or by Grade 2 or Grade 3 pyrophosphate (PYP) positivity (exception: nonamyloid control arm in aim 1).\n\n * Diagnosis of heart failure, with prior or current need of diuretics and increased N-terminal prohormone B-natureitic peptide (BNP) (≥450 pg/ml).\n * Peak VO2 \\<80% predicted, indicating impaired aerobic capacity (for aim 2 only).\n * Taking tafamidis (for aim 2 only)\n * Able to walk 4 meters (with or without the use of an assistive device) and independent with basic activities of daily living at the time of enrolment.\n * Adequate clinical stability has been achieved in the judgment of the investigator to allow participation in study assessments and the intervention.\n * Signed informed consent document indicating that the patient understands the purpose of and procedures required for the study and is willing to participate in the study.\n\nExclusion Criteria:\n\n* Acute myocardial infarction (Note: given that cardiac biomarkers such as troponin are frequently elevated in ATTR-CM patients, the diagnosis of acute myocardial infarction should be based on clinical diagnosis, not biomarkers alone)\n\n * \\>70% obstructive coronary artery disease\n * Severe aortic valve stenosis\n * Already actively participating in formal, facility-based cardiac exercise\n * Already engaging in regular moderate to vigorous exercise conditioning defined as \\> 30 minutes per day, ≥ twice per week consistently during the previous 6 weeks\n * Ventricular assist device\n * Light chain amyloidosis or other form of non-ATTR amyloidosis\n * Advanced chronic kidney disease defined as estimated glomerular filtration rate \\<20 mL/min/1.73m2\n * Any organ transplantation\n * Terminal illness other than HF with life expectancy \\< 1 year\n * Pacemaker or implantable cardioverter-defibrillato (ICD) with heart rate limits \\< expected heart rates for exercise and unable to be reprogrammed\n * Neuropathy due to transthyretin (TTR) mutation\n * Impairment from stroke, injury or other medical disorder that precludes participation in the intervention\n * Abnormal cardiopulmonary exercise testing (CPET) finding that requires further investigation and management\n * Dementia that precludes ability to participate in exercise and follow study protocols\n * High risk for non-adherence as determined by screening evaluation\n * Inability or unwillingness to comply with the study requirements'}, 'identificationModule': {'nctId': 'NCT05797857', 'briefTitle': 'Exercise Training in Transthyretin Cardiac Amyloidosis', 'organization': {'class': 'OTHER', 'fullName': "Brigham and Women's Hospital"}, 'officialTitle': 'Personalized Exercise Training to Improve Functional Capacity in Transthyretin Cardiac Amyloidosis', 'orgStudyIdInfo': {'id': '2022p002754'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Intervention arm', 'interventionNames': ['Other: Exercise Training']}], 'interventions': [{'name': 'Exercise Training', 'type': 'OTHER', 'description': 'A supervised, personalized exercise training program, which will consist of two 60-minute exercise training sessions per week.', 'armGroupLabels': ['Intervention arm']}]}, 'contactsLocationsModule': {'locations': [{'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Sarah Cuddy, MD', 'role': 'CONTACT', 'email': 'scuddy1@bwh.harvard.edu', 'phone': '6177328410'}], 'facility': "Brigham and Women's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': 'A limited access dataset generated from this project will rapidly be made available to the broader community for distribution to outside researchers according to established Mass General Brigham and NHLBI procedures and in accordance with NIH policies. Exercise training plans developed as part of this proposal will be made available.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': "Brigham and Women's Hospital", 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Cardiologist', 'investigatorFullName': 'Sarah Cuddy, MD', 'investigatorAffiliation': "Brigham and Women's Hospital"}}}}