Ignite Creation Date:
2025-12-24 @ 5:45 PM
Ignite Modification Date:
2025-12-26 @ 9:46 AM
Study NCT ID:
NCT07080268
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-07-23
First Post:
2025-07-15
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis.
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C567782', 'term': 'Amyloidosis, Hereditary, Transthyretin-Related'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 70}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2025-06-20', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-07', 'completionDateStruct': {'date': '2025-12-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-07-15', 'studyFirstSubmitDate': '2025-07-15', 'studyFirstSubmitQcDate': '2025-07-15', 'lastUpdatePostDateStruct': {'date': '2025-07-23', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-07-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-09-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To develop and validate a structured and self-administered quality of life questionnaire in patients with hereditary amyloidosis', 'timeFrame': '6 months', 'description': 'To develop and validate a structured and self-administered questionnaire specific to covering physical, emotional and social aspects, to assess the impact of hereditary transthyretin amyloidosis on the quality of life of patients residing in Argentina.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Amyloidosis in Transthyretin (TTR)', 'Hereditary Amyloidosis, Transthyretin-Related']}, 'referencesModule': {'references': [{'pmid': '31339362', 'type': 'BACKGROUND', 'citation': 'Conceicao I, Coelho T, Rapezzi C, Parman Y, Obici L, Galan L, Rousseau A. Assessment of patients with hereditary transthyretin amyloidosis - understanding the impact of management and disease progression. Amyloid. 2019 Sep;26(3):103-111. doi: 10.1080/13506129.2019.1627312. Epub 2019 Jul 24.'}, {'pmid': '33982288', 'type': 'BACKGROUND', 'citation': 'Aimo A, Rapezzi C, Perfetto F, Cappelli F, Palladini G, Obici L, Merlini G, Di Bella G, Serenelli M, Zampieri M, Milani P, Licordari R, Teresi L, Ribarich N, Castiglione V, Quattrone F, De Rosis S, Vergaro G, Panichella G, Emdin M, Passino C. Quality of life assessment in amyloid transthyretin (ATTR) amyloidosis. Eur J Clin Invest. 2021 Nov;51(11):e13598. doi: 10.1111/eci.13598. Epub 2021 May 22.'}]}, 'descriptionModule': {'briefSummary': 'Prospective study of development and validation of a questionnaire on quality of life in patients with Hereditary Amyloidosis The questionnaire development process will include 4 medical specialists experts in Transthyretin Amyloidosis (2 neurologists, 1 cardiologist, 1 physician clinical/hepathologist), 1 psychologist; 1 social worker; and 3 patients (with confirmed Hereditary amyloidosis) members of ALAPA. (Local PAG)\n\nPopulation and sample:'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients with confirmed ATTR (genetic test), older than 18 years in stage I, II and III of the PND (polyneuropathy disability) scale', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* patients with confirmed ATTR (genetic test), older than 18 years old in stage I, II and III of the PND scale (polyneuropathy disability: I Sensory neuropathy ; II Motor symptoms, but with preserved ambulation; IIIa Requires 1 cane to ambulate; IIIb requires two canes/walker).\n\nExclusion Criteria:\n\n* patients who participated in the validation process of the questionnaire: patients who participated in the questionnaire construction process or adjustments to the questionnaire.'}, 'identificationModule': {'nctId': 'NCT07080268', 'briefTitle': 'Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis.', 'organization': {'class': 'OTHER', 'fullName': 'Hospital General de Niños Pedro de Elizalde'}, 'officialTitle': 'Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis in Argentina.', 'orgStudyIdInfo': {'id': 'HGNPE'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Symptomatic patients with hereditary amyloidosis'}]}, 'contactsLocationsModule': {'locations': [{'zip': '1752', 'city': 'Buenos Aires', 'state': 'Buenos Aires', 'country': 'Argentina', 'facility': 'HGNPE'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Hospital General de Niños Pedro de Elizalde', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'PhD', 'investigatorFullName': 'Fernando Torres', 'investigatorAffiliation': 'Hospital General de Niños Pedro de Elizalde'}}}}