Ignite Creation Date:
2025-12-25 @ 1:10 AM
Ignite Modification Date:
2025-12-25 @ 11:21 PM
Study NCT ID:
NCT04943393
Status:
COMPLETED
Last Update Posted:
2022-04-22
First Post:
2021-06-02
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Remote Neurocognitive and Psychological Assessment in PKU
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D010661', 'term': 'Phenylketonurias'}], 'ancestors': [{'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000592', 'term': 'Amino Acid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITHOUT_DNA', 'description': 'Blood phe levels'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'CROSS_SECTIONAL', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 84}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-11-24', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-04', 'completionDateStruct': {'date': '2021-10-02', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-04-21', 'studyFirstSubmitDate': '2021-06-02', 'studyFirstSubmitQcDate': '2021-06-27', 'lastUpdatePostDateStruct': {'date': '2022-04-22', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-06-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-10-02', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Working Memory performance', 'timeFrame': '2.5-3 hrs total over approx 1-3 weeks', 'description': 'Composite score based on multiple working memory-related CANTAB tests'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Phenylketonurias']}, 'descriptionModule': {'briefSummary': 'PKU is a rare autosomal recessive condition associated with disruption in dopamine synthesis. Although early diagnosis and treatment prevent the severe impairments associated with untreated PKU, individuals with early-treated PKU (ETPKU) nonetheless experience significant sequelae, including impaired working memory and increased risk for anxiety problems. Past research with other clinical populations (e.g., generalized anxiety disorder) suggests that the two phenomena may be linked, with increased anxiety contributing to poorer WM performance. Currently we propose to initiate an exciting new line of research examining the potential relationship between anxiety and WM in ETPKU. The proposed study will also apply remote neuropsychological performance-based assessment to the study of ETPKU and thus overcoming the geographical limitations/challenges associated with recruitment of participants with this rare disease.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '40 Years', 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Individuals age 18-40 years old with and without PKU', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participants with PKU will be included if they were diagnosed as newborns and immediately started on treatment.\n\nExclusion Criteria:\n\n* Individuals with a history of neurologic compromise or major medical disorder unrelated to PKU (e.g., closed head injury, diabetes, multiple sclerosis) will be excluded. In addition, individuals with PKU will be excluded if they are on a medication called Peg-Valiant/ PEG-PAL/ Pegvaliase/Palynziq.'}, 'identificationModule': {'nctId': 'NCT04943393', 'briefTitle': 'Remote Neurocognitive and Psychological Assessment in PKU', 'organization': {'class': 'OTHER', 'fullName': 'University of Missouri-Columbia'}, 'officialTitle': 'Remote Assessment of Working Memory and Anxiety in Individuals With Early-Treated Phenylketonuria (PKU)', 'orgStudyIdInfo': {'id': '2034504'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Individuals with PKU', 'description': 'Adults with early-treated PKU', 'interventionNames': ['Other: nointervention']}, {'label': 'Individuals without PKU', 'description': 'Adults without PKU who are otherwise healthy', 'interventionNames': ['Other: nointervention']}], 'interventions': [{'name': 'nointervention', 'type': 'OTHER', 'description': 'Nointervention', 'armGroupLabels': ['Individuals with PKU', 'Individuals without PKU']}]}, 'contactsLocationsModule': {'locations': [{'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '65211', 'city': 'Columbia', 'state': 'Missouri', 'country': 'United States', 'facility': 'University of Missouri', 'geoPoint': {'lat': 38.95171, 'lon': -92.33407}}], 'overallOfficials': [{'name': 'Shawn Christ, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Missouri-Columbia'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED', 'description': 'To be determined'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Missouri-Columbia', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Associate Professor', 'investigatorFullName': 'Shawn Christ', 'investigatorAffiliation': 'University of Missouri-Columbia'}}}}