Ignite Creation Date:
2025-12-25 @ 12:02 AM
Ignite Modification Date:
2026-04-24 @ 4:12 AM
Study NCT ID:
NCT02587858
Status:
UNKNOWN
Last Update Posted:
2020-10-08
First Post:
2015-10-23
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
NBIAready: Online Collection of Natural History Patient-reported Outcome Measures
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006211', 'term': 'Pantothenate Kinase-Associated Neurodegeneration'}, {'id': 'D019150', 'term': 'Neuroaxonal Dystrophies'}], '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': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'BPAN participants will be asked for blood and saliva samples.'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 300}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2015-04'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-10', 'completionDateStruct': {'date': '2025-04', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2020-10-05', 'studyFirstSubmitDate': '2015-10-23', 'studyFirstSubmitQcDate': '2015-10-23', 'lastUpdatePostDateStruct': {'date': '2020-10-08', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2015-10-27', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2025-04', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Document the natural history of NBIA and identify new markers of disease progression.', 'timeFrame': '5-10 years', 'description': 'The unit of analyses will be the data collected on NBIA milestones and patient-reported outcome measures. Latent Growth Curve (LGC) modeling will be used to characterize the natural history of NBIA. If data support that certain disease milestones occur in a consistent order, or at consistent ages, then these data points will be candidate markers of disease progression. LGC will be used to characterize the natural history of NBIA and identify areas in which individuals with NBIA differ from the general population.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['NBIA', 'PKAN', 'brain iron', 'Neurodegeneration with Brain Iron Accumulation', 'Hallervorden Spatz Syndrome', 'Pantothenate Kinase Associated Neurodegeneration', 'PLAN', 'INAD', 'PLA2G6-associated neurodegeneration', 'BPAN', 'Beta-propeller Protein-associated neurodegenration', 'WDR45', 'PLA2G6'], 'conditions': ['Neurodegeneration With Brain Iron Accumulation (NBIA)']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.nbiacure.org', 'label': 'Official website of the OHSU NBIA research group'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to learn more about Neurodegeneration with Brain Iron Accumulation (NBIA) Disorders. Data is being collected on three types of NBIA disorders:\n\nPantothenate Kinase-Associated Neurodegeneration (PKAN), PLA2G6-associated Neurodegeneration (PLAN) and Beta-propeller Protein-associated Neurodegeneration (BPAN). The study will (1) collect information about how symptoms and findings in NBIA change over time and (2) identify measures of NBIA that can be used in future clinical trials. Participants will follow links to a secure website every 6 months for a period of 5-10 years to electronically complete a set of rating scales as related to their NBIA disorder.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'The PKAN group consists of international individuals diagnosed with PKAN. The PLAN group consists of international individuals diagnosed with PLAN. The BPAN group consists of international individuals diagnosed with BPAN.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of PKAN, PLAN or BPAN confirmed by gene testing and/or clinical features.\n* Ability to access a computer with internet services or a phone approximately once every 6 months for up to 10 years to enter data.\n\nExclusion Criteria:\n\n* Individuals who are not fluent in reading and communicating in English.'}, 'identificationModule': {'nctId': 'NCT02587858', 'briefTitle': 'NBIAready: Online Collection of Natural History Patient-reported Outcome Measures', 'organization': {'class': 'OTHER', 'fullName': 'Oregon Health and Science University'}, 'officialTitle': 'NBIAready: Online Collection of Natural History Patient-reported Outcome Measures', 'orgStudyIdInfo': {'id': '10832'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'PKAN', 'description': 'This group consists of individuals diagnosed with PKAN using a combination of MRI, other clinical findings, and PANK2 gene sequencing.'}, {'label': 'PLAN', 'description': 'This group consists of individuals diagnosed with PLAN using a combination of MRI, other clinical findings and PLA2G6 gene sequencing.'}, {'label': 'BPAN', 'description': 'This group consists of individuals diagnosed with BPAN using a combinatino of MRI, other clinical findings, and WDR45 gene sequencing.'}]}, 'contactsLocationsModule': {'locations': [{'zip': '97239-3098', 'city': 'Portland', 'state': 'Oregon', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Alison C Freed, BA', 'role': 'CONTACT', 'email': 'freeal@ohsu.edu', 'phone': '503-494-6838'}, {'name': 'Allison M Gregory, MS, CGC', 'role': 'CONTACT', 'email': 'gregorya@ohsu.edu', 'phone': '503 494 4344'}, {'name': 'Susan J Hayflick, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Oregon Health & Science University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}], 'centralContacts': [{'name': 'Alison C Freed, BA', 'role': 'CONTACT', 'email': 'freeal@ohsu.edu', 'phone': '503 494 6838'}, {'name': 'Allison M Gregory, MS, CGC', 'role': 'CONTACT', 'email': 'gregorya@ohsu.edu', 'phone': '503 494 4344'}], 'overallOfficials': [{'name': 'Susan J Hayflick, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Oregon Health and Science University'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Susan J. Hayflick', 'class': 'OTHER'}, 'collaborators': [{'name': 'Oregon Health and Science University', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Professor & Chair, Molecular & Medical Genetics', 'investigatorFullName': 'Susan J. Hayflick', 'investigatorAffiliation': 'Oregon Health and Science University'}}}}