Ignite Creation Date:
2025-12-26 @ 2:43 PM
Ignite Modification Date:
2025-12-26 @ 2:43 PM
Study NCT ID:
NCT04210206
Status:
TERMINATED
Last Update Posted:
2023-12-14
First Post:
2019-12-19
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria
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': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 13}}, 'statusModule': {'whyStopped': 'Recruitment failure', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2019-12-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-12', 'completionDateStruct': {'date': '2023-08-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-12-08', 'studyFirstSubmitDate': '2019-12-19', 'studyFirstSubmitQcDate': '2019-12-20', 'lastUpdatePostDateStruct': {'date': '2023-12-14', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-12-24', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-08-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Spatial working memory strategy index', 'timeFrame': '8 weeks', 'description': 'cognitive function; scores range from 4 to 28, with lower scores representing better outcome'}], 'secondaryOutcomes': [{'measure': 'Reaction time', 'timeFrame': '8 weeks', 'description': 'cognitive function; milliseconds, with faster times representing better outcome'}, {'measure': 'Paired Associates Learning', 'timeFrame': '8 weeks', 'description': 'cognitive function; scores range from 0 to 70, with lower scores indicating better outcome'}, {'measure': 'Multitasking Test', 'timeFrame': '8 weeks', 'description': 'cognitive function; scores range from 0 to 279, with lower scores indicating better outcome'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['cognitive function adults'], 'conditions': ['Phenylketonurias']}, 'descriptionModule': {'briefSummary': 'The goal of this study is to evaluate if in adult patients with phenylketonuria (PKU) without strict metabolic control during the last 12 months, strict metabolic control for 8 weeks results in an improvement of cognitive function.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '59 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Men and women with classical PKU\n* Age ≥ 18 years\n* Good metabolic control during childhood (at least until 12 years of age)\n* Plasma Phe levels ≥ 15 mg/dl for ≥ 1 year\n* Written informed consent prior to study participation\n\nExclusion Criteria:\n\n* Reduced cognitive function (unability to perform test battery)\n* Drug and or alcohol abuse\n* Treatment with BH4\n* Treatment with drugs known to affect cognitive function'}, 'identificationModule': {'nctId': 'NCT04210206', 'briefTitle': 'Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria', 'organization': {'class': 'OTHER', 'fullName': 'Ludwig-Maximilians - University of Munich'}, 'officialTitle': 'Kurzfristiger Einfluss Einer Guten Stoffwechseleinstellung Auf Kognitive Funktion, Wohlbefinden Und Stoffwechselparameter Bei Erwachsenen Patienten Mit Phenylketonurie (Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria)', 'orgStudyIdInfo': {'id': 'CoFu-PKU'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Diet', 'interventionNames': ['Other: PHE-diet']}, {'type': 'NO_INTERVENTION', 'label': 'Control'}], 'interventions': [{'name': 'PHE-diet', 'type': 'OTHER', 'description': 'phenylalanine-restricted diet with phenylalanine free supplements for 8 weeks', 'armGroupLabels': ['Diet']}]}, 'contactsLocationsModule': {'locations': [{'zip': '81377', 'city': 'Munich', 'country': 'Germany', 'facility': 'University Munich', 'geoPoint': {'lat': 48.13743, 'lon': 11.57549}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Klaus Parhofer', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Professor of Endocrinology and Metabolism', 'investigatorFullName': 'Klaus Parhofer', 'investigatorAffiliation': 'Ludwig-Maximilians - University of Munich'}}}}