Ignite Creation Date:
2025-12-24 @ 10:32 PM
Ignite Modification Date:
2025-12-25 @ 8:05 PM
Study NCT ID:
NCT04691635
Status:
UNKNOWN
Last Update Posted:
2022-02-11
First Post:
2020-12-28
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
The MOTIVE-PSP Initiative (Progressive Supranuclear Palsy)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D013494', 'term': 'Supranuclear Palsy, Progressive'}], 'ancestors': [{'id': 'D001480', 'term': 'Basal Ganglia Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D009069', 'term': 'Movement Disorders'}, {'id': 'D009886', 'term': 'Ophthalmoplegia'}, {'id': 'D015835', 'term': 'Ocular Motility Disorders'}, {'id': 'D003389', 'term': 'Cranial Nerve Diseases'}, {'id': 'D024801', 'term': 'Tauopathies'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D010243', 'term': 'Paralysis'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'CASE_CONTROL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 165}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2022-01-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-01', 'completionDateStruct': {'date': '2025-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2022-01-27', 'studyFirstSubmitDate': '2020-12-28', 'studyFirstSubmitQcDate': '2020-12-28', 'lastUpdatePostDateStruct': {'date': '2022-02-11', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-12-31', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Progressive Supranuclear Palsy rating scale', 'timeFrame': '1-year follow up', 'description': 'Progression of disease as assessed with the standard clinical scale rating (scoring 0-100, higher scores indicate worse scores) Progressive Supranuclear Palsy symptoms and signs'}], 'secondaryOutcomes': [{'measure': 'Cognitive progression', 'timeFrame': '1-year follow up', 'description': 'Evaluated with the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) scale score (scoring 0-100, higher scores indicate better scores)'}, {'measure': 'Behavioral progression', 'timeFrame': '1-year follow up', 'description': 'Behavioral symptoms as assessed with Neuropsychiatric Inventory scale (scoring 0-144, higher scores indicate worse scores)'}, {'measure': 'MRI', 'timeFrame': '1-year follow up', 'description': 'Progression of disease as assessed with MRI specific measures (the superior cerebellar peduncles, the middle cerebellar peduncles, the MR Parkinsonism index, the midbrain-pons area ratio; a 3D Spoiled Gradient Recalled Echo (SPGR) and a 3D-GRE multi-echo susceptibility weighted)'}, {'measure': 'Gait speed', 'timeFrame': '1-year follow up', 'description': 'Gait speed as assessed with motion sensors'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Progressive Supranuclear Palsy']}, 'referencesModule': {'references': [{'pmid': '33281738', 'type': 'BACKGROUND', 'citation': 'Picillo M, Abate F, Ponticorvo S, Tepedino MF, Erro R, Frosini D, Del Prete E, Cecchi P, Cosottini M, Ceravolo R, Salle GD, Salle FD, Esposito F, Pellecchia MT, Manara R, Barone P. Association of MRI Measures With Disease Severity and Progression in Progressive Supranuclear Palsy. Front Neurol. 2020 Nov 12;11:603161. doi: 10.3389/fneur.2020.603161. eCollection 2020.'}, {'pmid': '31603934', 'type': 'BACKGROUND', 'citation': 'Picillo M, Cuoco S, Carotenuto I, Abate F, Erro R, Volpe G, Pellecchia MT, Catricala E, Cappa S, Barone P. Clinical use of SAND battery to evaluate language in patients with Progressive Supranuclear Palsy. PLoS One. 2019 Oct 11;14(10):e0223621. doi: 10.1371/journal.pone.0223621. eCollection 2019.'}, {'pmid': '31527182', 'type': 'BACKGROUND', 'citation': 'Picillo M, Tepedino MF, Abate F, Erro R, Ponticorvo S, Tartaglione S, Volpe G, Frosini D, Cecchi P, Cosottini M, Ceravolo R, Esposito F, Pellecchia MT, Barone P, Manara R. Midbrain MRI assessments in progressive supranuclear palsy subtypes. J Neurol Neurosurg Psychiatry. 2020 Jan;91(1):98-103. doi: 10.1136/jnnp-2019-321354. Epub 2019 Sep 16.'}, {'pmid': '31350659', 'type': 'BACKGROUND', 'citation': 'Picillo M, Cuoco S, Amboni M, Bonifacio FP, Bruschi F, Carotenuto I, De Micco R, De Rosa A, Del Prete E, Di Biasio F, Elifani F, Erro R, Fabbri M, Falla M, Franco G, Frosini D, Galantucci S, Lazzeri G, Magistrelli L, Malaguti MC, Milner AV, Minafra B, Olivola E, Pilotto A, Rascuna C, Rizzetti MC, Schirinzi T, Borroni B, Ceravolo R, Di Fonzo A, Marchese R, Mercuri NB, Modugno N, Nicoletti A, Padovani A, Santangelo G, Stefani A, Tessitore A, Volonte MA, Zangaglia R, Zappia M, Zibetti M, Barone P. Validation of the Italian version of the PSP Quality of Life questionnaire. Neurol Sci. 2019 Dec;40(12):2587-2594. doi: 10.1007/s10072-019-04010-2. Epub 2019 Jul 26.'}, {'pmid': '31190253', 'type': 'BACKGROUND', 'citation': "Picillo M, Cuoco S, Amboni M, Bonifacio FP, Bruno A, Bruschi F, Cappiello A, De Micco R, De Rosa A, Di Biasio F, Elifani F, Erro R, Fabbri M, Falla M, Franco G, Frosini D, Galantucci S, Lazzeri G, Magistrelli L, Malaguti MC, Milner AV, Minafra B, Olivola E, Pilotto A, Rascuna C, Rizzetti MC, Schirinzi T, Borroni B, Ceravolo R, Di Fonzo A, Lopiano L, Marchese R, Mercuri NB, Modugno N, Nicoletti A, Padovani A, Santangelo G, Stefani A, Tessitore A, Volonte MA, Zangaglia R, Zappia M, Barone P. Validation of the Italian version of carers' quality-of-life questionnaire for parkinsonism (PQoL Carer) in progressive supranuclear palsy. Neurol Sci. 2019 Oct;40(10):2163-2169. doi: 10.1007/s10072-019-03944-x. Epub 2019 Jun 12."}, {'pmid': '30989369', 'type': 'BACKGROUND', 'citation': 'Picillo M, Cuoco S, Tepedino MF, Cappiello A, Volpe G, Erro R, Santangelo G, Pellecchia MT, Barone P; PSP Salerno study group. Motor, cognitive and behavioral differences in MDS PSP phenotypes. J Neurol. 2019 Jul;266(7):1727-1735. doi: 10.1007/s00415-019-09324-x. Epub 2019 Apr 15.'}, {'pmid': '29984518', 'type': 'BACKGROUND', 'citation': 'Picillo M, Erro R, Cuoco S, Tepedino MF, Manara R, Pellecchia MT, Barone P; PSP Salerno Study Group. MDS PSP criteria in real-life clinical setting: Motor and cognitive characterization of subtypes. Mov Disord. 2018 Aug;33(8):1361-1365. doi: 10.1002/mds.27408. Epub 2018 Jul 8. No abstract available.'}]}, 'descriptionModule': {'briefSummary': 'Progressive Supranuclear Palsy (PSP) is a rapidly progressive neurodegenerative disease characterized by falls and oculomotor disturbances. Several clinical trials are currently evaluating the efficacy of new pharmacological compounds in slowing disease progression. Yet, both early diagnosis and evaluation of disease progression remain challenging.\n\nStudy aims include verifying if specific motor, cognitive, language, cerebrospinal fluid and imaging assessments represent reliable biomarkers of PSP diagnosis, phenotypization and progression over 1-year follow up. Motor evaluation will include recordings from wearable sensors.\n\nExpected results include 1) improvement of PSP diagnosis and phenotypization; 2)improvement of evaluation of disease progression in the context of clinical trial; 3)enhancement of strategies to prevent falls and fractures in such patients leading, in turn, to significant cost savings for the National Health System.', 'detailedDescription': "Hyphotesis and Significance:\n\n1)Cross-sectional phase:verify if specific motor, cognitive, language, cerebrospinal fluid (CSF) and imaging assessments represent reliable biomarkers of diagnosis in PSP in the earliest stages of disease compared to Parkinson's disease (PD) and healthy controls (HC) and are able characterize the different PSP clinical phenotype 2)12-month longitudinal phase:verify if specific motor, cognitive, language, CSF and imaging assessments represent reliable biomarkers of disease progression also according to the clinical phenotype\n\nPreliminary Data:\n\nCross-sectional data suggest that current motor, cognitive, language and imaging assessments may be useful in supporting the diagnosis of PSP compared to PD and HC, but not in characterizing the PSP clinical phenotypes. No robust data on wearable sensors and CSF biomarkers is available.\n\nLongitudinal data on such biomarkers are lacking. As for cognition, preliminary data suggest that the Repeatable Battery for the assessment of neuropsychological status (RBANS) may evaluate cognitive trajectories in PSP with Richardson's syndrome.\n\nSpecific Aim 1:\n\nDetermine the feasibility, validity and reliability of application of digital biomarkers in PSP. Specifically if clinic-based wearable sensors and smartphone-based remote assessments can (1) support early clinical diagnosis and phenotyping of PSP patients compared to PD and HC and (2) evaluate disease trajectories in the whole cohort of PSP patients and discrete phenotypes compared to standard rating scales.\n\nFor both points the hypothesis is that both clinic-based and smartphone-based wearable sensors outperform standard rating scales.\n\nSpecific aim 2:\n\nVerify if cognitive, language and behavioral testing can (1) support early clinical diagnosis and phenotyping of PSP patients compared to PD and HC and (2) evaluate disease trajectories in the whole cohort of PSP patients and discrete phenotypes.\n\nFor point (1) the hypothesis is that cognitive, language and behavioral testing support early diagnosis and phenotyping in the earliest stages compared to PD and HC. For point (2) the hypothesis is that cognitive, language and behavioral testing are able to evaluate disease progression in the whole cohort of PSP patients and discrete phenotypes.\n\nSpecific aim 3:\n\nVerify if single parameter and multiparameter magnetic resonance imaging (MRI) imaging assessments can (1) support early clinical diagnosis and phenotyping of PSP patients compared to PD and HC and (2) evaluate disease trajectories in the whole cohort of PSP patients and discrete phenotypes.\n\nExperimental design aim 1 includes a cross-sectional phase with enrollment of 3 groups of subjects (PSP, PD and HC).\n\nDuring the baseline evaluation each enrolled subject will perform:1) a clinic-based standardized protocol for assessment of gait with wearable inertial sensors to specifically monitor quality of gait and balance using a wide range of measures from the upper and lower body (APDM Mobility Lab system) ;2) home-based monitoring with smartphone of inertial measurement (balance and gait task) and voice recorded with microphone for at least 5 days;3) standard clinical rating scales including the PSP rating scale, the Natural History and Neuroprotection in Parkinson Plus Syndromes, Movement Disorder Society Unified Parkinson's disease rating scale part III, Falls diary to be filled during the 5-day home based monitoring. Caregivers will be asked to actively support patients for the home- smartphone-based digital biomarkers measurements.\n\nFurthermore, at baseline only PSP patients will be asked to perform lumbar puncture to collect CSF and measure total-tau, phosphorylated tau, Beta-amyloid 42, neurofilament light and heavy chain. A 12-month longitudinal phase will follow only for PSP patients.\n\nDuring both cross-sectional and longitudinal phases (every 3 months), only patients enrolled at Unit 1 will also perform a gait analysis assessment with a traditional opto-kinematic, hospital-based gait analysis system (Qualisys®, Sandvälen, Sweden).\n\nExperimental design aim 2 includes a cross-sectional phase with enrollment of 3 groups of subjects (PSP, PD and HC).\n\nDuring the baseline evaluation each enrolled subject will perform:1) the RBANS; 2)standard cognitive assessment currently used by part of the research team to evaluate cognitive abilities in PSP patients as detailed elsewhere; 3) functional autonomy will be evaluated with the Instrumental Activities of Daily Life, while depression and apathy with the Beck Depression Inventory II and Apathy Evaluation Scale, respectively; 4) other neuropsychiatric symptoms will be explored with the Neuropsychiatric Inventory; 5) language abilities will be explored with the Screening for Aphasia in NeuroDegeneration (SAND) battery.\n\nA 12-month longitudinal phase will follow only for PSP patients.\n\nExperimental design aim 3 includes a cross-sectional phase with enrollment of 3 groups of subjects (PSP, PD and HC).\n\nDuring the baseline evaluation each enrolled subject will undergo a multimodal 3T MRI. The MRI protocol will include conventional sequences for morphometric measures (ie, the superior cerebellar peduncles, the middle cerebellar peduncles, the MR Parkinsonism index, the midbrain-pons area ratio; a 3D Spoiled Gradient Recalled Echo (SPGR) and a 3D-GRE multi-echo susceptibility weighted (SWAN) images to obtain quantitative susceptibility mapping and ROI based measures in substantia nigra, basal ganglia thalamus, red nucleus and fronto-parietal cortex, a resting state functional MRI and an Arterial Spin Labeling sequence.\n\nA 12-month longitudinal phase will follow only for PSP patients. Both single parameter and multiparameter (morphometric, iron based, functional, and perfusion) MRI approaches will be applied to validate the usefulness of imaging assessments in predicting both early PSP diagnosis and phenotyping as well as disease progression."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '40 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients with Progressive Supranuclera palsy. Any phenotype of the disease will be included', 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of Progressive Supranuclear Palsy based on current available clinical criteria\n* ability to walk for at least 5 steps\n\nExclusion Criteria:\n\n* Comorbidities interfering with study assessments\n* Significant MRI abnormalities as cerebrovascular diseases, tumors.'}, 'identificationModule': {'nctId': 'NCT04691635', 'briefTitle': 'The MOTIVE-PSP Initiative (Progressive Supranuclear Palsy)', 'organization': {'class': 'OTHER', 'fullName': 'University of Salerno'}, 'officialTitle': 'MotOr, cogniTIVe and Imaging charactErization of Progressive Supranuclear Palsy Phenotypes: a Longitudinal Prospective Study Looking for Biomarkers', 'orgStudyIdInfo': {'id': 'CEMAND-2020-02'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Group 1', 'description': 'Patients with Progressive Supranuclear Palsy', 'interventionNames': ['Other: evaluation of movements with sensors']}, {'label': 'Group 2', 'description': "Patients with Parkinson's disease", 'interventionNames': ['Other: evaluation of movements with sensors']}, {'label': 'Group 3', 'description': 'Healthy controls', 'interventionNames': ['Other: evaluation of movements with sensors']}], 'interventions': [{'name': 'evaluation of movements with sensors', 'type': 'OTHER', 'description': 'evaluation of movements with sensors', 'armGroupLabels': ['Group 1', 'Group 2', 'Group 3']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Padua', 'status': 'NOT_YET_RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Miryam Carecchio, MD', 'role': 'CONTACT', 'email': 'padovaparkinson@gmail.com'}], 'facility': 'AOU Padova', 'geoPoint': {'lat': 45.40797, 'lon': 11.88586}}, {'city': 'Pisa', 'status': 'NOT_YET_RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Daniela Frosini, MD', 'role': 'CONTACT', 'email': 'daniela.frosini@ao-pisa.toscana.it', 'phone': '0039050993213'}, {'name': 'Eleonora Del Prete, MD', 'role': 'CONTACT', 'email': 'amb.parkinsonpisa@ao-pisa.toscana.it'}], 'facility': 'AOU Pisa', 'geoPoint': {'lat': 43.70853, 'lon': 10.4036}}, {'zip': '84131', 'city': 'Salerno', 'status': 'RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Marina Picillo, MD', 'role': 'CONTACT', 'email': 'mpicillo@unisa.it', 'phone': '3497725402'}, {'name': 'Filomena Abate, MD', 'role': 'CONTACT', 'email': 'fi.abate@hotmail.it'}], 'facility': "AOU San Giovanni di Dio e Ruggi d'Aragona", 'geoPoint': {'lat': 40.67545, 'lon': 14.79328}}], 'centralContacts': [{'name': 'Marina Picillo, MD', 'role': 'CONTACT', 'email': 'mpicillo@unisa.it', 'phone': '00393497725402'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': 'No IPD will be shared with external researchers'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Salerno', 'class': 'OTHER'}, 'collaborators': [{'name': 'Azienda Ospedaliera di Padova', 'class': 'OTHER'}, {'name': 'Azienda Ospedaliero, Universitaria Pisana', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Doctor', 'investigatorFullName': 'Marina Picillo', 'investigatorAffiliation': 'University of Salerno'}}}}