protocolSection identificationModule nctId: NCT06365593, orgStudyIdInfo id: D8450R00005, briefTitle: hATTR Polyneuropathy in Russia, acronym: PRIMER, statusModule overallStatus: RECRUITING, startDateStruct date: 2023-12-29, primaryCompletionDateStruct date: 2024-05-31, completionDateStruct date: 2024-05-31, studyFirstPostDateStruct date: 2024-04-15, lastUpdatePostDateStruct date: 2024-04-15, sponsorCollaboratorsModule leadSponsor name: AstraZeneca, class: INDUSTRY, descriptionModule briefSummary: A multicenter observational retrosPective Registry of patIents with transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic idiopathic axonal polyneuropathy (CIAP) in the population of the Russian Federation (PRIMER) There are no comprehensive epidemiological data on patients with hereditary ATTR-PN (hATTR-PN) and CIAP in the Russian Federation. Therefore, there is a need to conduct a large-scale observational study in the Russian population to obtain information on clinical, electrophysiological and demographic characteristics of patients with hATTR-PN and CIAP. Obtaining the study data will help to identify the patients with axonal polyneuropathy, who deserve TTR gene sequencing, and therefore to allow early treatment and potentially modify disease progression in patients., conditionsModule conditions: Hereditary Transthyretin Amyloidosis, designModule studyType: OBSERVATIONAL, designInfo observationalModel: COHORT, timePerspective: RETROSPECTIVE, enrollmentInfo count: 100, type: ESTIMATED, armsInterventionsModule, outcomesModule primaryOutcomes measure: In order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Mean age at the hATTR-PN or CIAP diagnosis information will be collected, primaryOutcomes measure: In order to describe the baseline demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Proportion of women and men will be collected, primaryOutcomes measure: In order to describe the baseline demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Proportion of patients with each race and ethnicity will be collected, primaryOutcomes measure: to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIA, information on Proportion of patients from each federal district of the Russian Federation will be collected, primaryOutcomes measure: in order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, tha following Anthropometric characteristics will be collected:, primaryOutcomes measure: In order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with underweight will be collected, primaryOutcomes measure: in order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Proportion of patients with negative lifestyle factors will be collected:, primaryOutcomes measure: in order to describe the baseline demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Proportion of patients with positive family history in close relatives will be collected, primaryOutcomes measure: to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP, information on Mean (or median) time from the first symptoms of neuropathy to the diagnosis of PN will be collected, primaryOutcomes measure: to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP, Proportion of patients with different firstly suspected etiology of PN will be calculated, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP, information on Mean (or median) time from first PN manifestation/diagnosis to the hATTR-PN or CIAP diagnosis will be collected, primaryOutcomes measure: To describe the baseline electrophysiological and clinical characteristics of patients, Proportion of patients with chronic sensory or sensorimotor polyneuropathy and progressive chronic polyneuropathy among patients with CIAP will be calculated, primaryOutcomes measure: To describe the baseline clinical characteristics of patients with hATTR-PN and CIAP, Proportion of patients with each result of TTR gene testing will be calculated, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with each clinical manifestation will be calculated:, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with each PND score will be calculated, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with each score by each parameter of neurological examination will be calculated, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean point by MRS will be calculated, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean point by INCAT scale (total score, arm disability, leg disability) will be collected, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean left ventricular ejection fraction (LVEF) will be collected, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information Mean wall thickness of the left ventricular (LV) will be collected, primaryOutcomes measure: To describe the baseline electrophysiological characteristics of patients with hATTR-PN and CIAP, information Mean variables based on the results of electrophysiological investigation, including the following (on both extremities) will be collected, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with diagnosis of HFpEF will be calculated, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean NT-proBNP level will be collected, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information Mean estimated glomerular filtration rate (eGFR) will be collected, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean urine albumin-to-creatinine ratio (UACR) in a spot urine sample will be collected, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with a positive paraprotein blood test result will be calculated, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean blood pressure (BP) (systolic and diastolic) and Mean heart rate (HR), primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with each concomitant disease will be calculated, primaryOutcomes measure: To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients underwent surgery for CTS to the moment of diagnosis will be calculated, secondaryOutcomes measure: To arrange a score to select patients with axonal neuropathy who deserve screening for TTR mutation., otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP in the Russian Federation during retrospective follow-up period Mean changes in BMI at visits 2, 3 will be compared to baseline, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Proportion of patients with newly diagnosed clinical manifestations will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period, Proportion of patients with different newly diagnosed clinical manifestations will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Proportion of patients with each PND score at visits 2, 3 will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP, Proportion of patients with worsening of PND score at visits 2, 3 compared to baseline will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Proportion of patients with hospitalisations (one or more) will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Proportion of different reasons for hospitalisations will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Proportion of patients with death for all causes will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients during follow-up period Proportion of patients with different causes of death will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients during retrospective follow-up period Proportion of patients with each score by each parameter of neurological examination at visits 2, 3 will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Mean changes in MRS at visits 2, 3 compared to baseline will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Mean changes in INCAT scale at visits 2, 3 compared to baseline will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Proportion of patients with diagnosis of HFpEF at visits 2, 3 will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Mean changes in LVEF at visits 2, 3 compared to baseline will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Mean changes in wall thickness of the LV at visits 2, 3 compared to baseline will be calculated, otherOutcomes measure: To assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Mean changes at visits 2, 3 compared to baseline will be calculated, otherOutcomes measure: to assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Mean changes in NT-proBNP at visits 2, 3 compared to baseline will be calculated, otherOutcomes measure: to assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Mean changes in eGFR at visits 2, 3 compared to baseline will be calculated, otherOutcomes measure: to assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Mean changes in UACR at visits 2, 3 compared to baseline will be calculated, otherOutcomes measure: to assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Proportion of patients with a positive paraprotein blood test result at visits 2, 3 will be calculated, otherOutcomes measure: to assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Mean changes in BP at visits 2, 3 compared to baseline will be calculated, otherOutcomes measure: to assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Mean changes in HR at visits 2, 3 compared to baseline will be calculated, otherOutcomes measure: to assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Proportion of patients with new concomitant diseases will be calculated, otherOutcomes measure: to assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Proportion of patients with different new concomitant diseases will be calculated, otherOutcomes measure: to assess the changes in the clinical and electrophysiological characteristics of patients with hATTR-PN and CIAP during retrospective follow-up period Proportion of patients with presence of CTS surgery will be calculated, eligibilityModule sex: ALL, stdAges: CHILD, stdAges: ADULT, stdAges: OLDER_ADULT, contactsLocationsModule locations facility: Research Site, status: RECRUITING, city: Kazan, country: Russian Federation, geoPoint lat: 55.78874, lon: 49.12214, locations facility: Research Site, status: RECRUITING, city: Moscow, country: Russian Federation, geoPoint lat: 55.75222, lon: 37.61556, locations facility: Research Site, status: RECRUITING, city: Saint-Petersburg, zip: 194354, country: Russian Federation, geoPoint lat: 59.93863, lon: 30.31413, hasResults: False