Ignite Creation Date:
2025-12-24 @ 7:36 PM
Ignite Modification Date:
2026-01-02 @ 9:59 AM
Study NCT ID:
NCT03856203
Status:
COMPLETED
Last Update Posted:
2023-11-18
First Post:
2019-02-14
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'submissionInfos': [{'resetDate': '2024-09-27', 'releaseDate': '2024-06-17'}], 'estimatedResultsFirstSubmitDate': '2024-06-17'}}, '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': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 12}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-03-08', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-11', 'completionDateStruct': {'date': '2023-10-20', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-11-15', 'studyFirstSubmitDate': '2019-02-14', 'studyFirstSubmitQcDate': '2019-02-25', 'lastUpdatePostDateStruct': {'date': '2023-11-18', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-02-27', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-12-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Intra-subject change in intact and medical food protein intake (grams/day) as reported on three-day food records at months 0, 9, and 15.', 'timeFrame': '15 months'}, {'measure': 'Intra-subject change in serum markers of protein status at months 0, 9, and 15.', 'timeFrame': '15 months'}, {'measure': 'Intra-subject difference in lean body mass (%LBM) from month 0 to 15.', 'timeFrame': '15 months'}, {'measure': 'Intra-subject difference in bone mineral density from month 0 to 15.', 'timeFrame': '15 months'}], 'secondaryOutcomes': [{'measure': 'Intra-subject change in concentrations of serum markers of micronutrient status at months 0, 9, and 15.', 'timeFrame': '15 months'}, {'measure': 'Intra-subject change in serum concentrations of essential fatty acid nutriture at months 0, 9, and 15.', 'timeFrame': '15 months'}, {'measure': 'Intra-subject change in BMI at months 0, 9, and 15.', 'timeFrame': '15 months'}, {'measure': 'Intra-subject change in serum markers of cardiovascular status at months 0, 9, and 15.', 'timeFrame': '15 months'}, {'measure': 'Intra-subject change in emotional eating, cognitive restraint, uncontrolled eating at months 0, 9, and 15.', 'timeFrame': '15 months', 'description': 'Three-Factor Eating Questionnaire (TFEQ)-18 is an 18-item questionnaire measured on a four-point scale cognitive restraint (6 items), uncontrolled eating (9 items) and emotional eating (3 items). Responses are measured on a four-point scale and the raw scores for each subscale are transformed to correspond to a 0-100 point scale. Higher scores in the respective scales are indicative of greater cognitive restraint, emotional eating, and uncontrolled eating.'}, {'measure': 'Intra-subject change in food neophobia at months 0, 9, and 15.', 'timeFrame': '15 months', 'description': 'Food Neophobia Scale, which was adapted for a PKU population, includes nine questions measured on a seven-point scale with a higher total score indicative of greater food neophobia (score range 9-63).'}, {'measure': 'Intra-subject change in Epicurean eating tendencies at months 0, 9, and 15.', 'timeFrame': '15 months', 'description': 'Epicurean eating tendencies questionnaire includes seven items measured with a seven-point scale (range 7-49). Higher scores indicative of greater Epicurean eating tendencies.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Phenylketonurias']}, 'descriptionModule': {'briefSummary': 'Conduct a prospective, longitudinal study to evaluate nutritional status in adults with phenylketonuria (PKU) before and during treatment with pegvaliase (Palynziq™).', 'detailedDescription': 'The investigators will compare intra-subject change in adults with PKU who follow a protein-restricted diet (protein intake less than the RDA for protein based on weight (0.8 g/kg/day) with or without a PKU medical food) within 90 days of starting pegvaliase (baseline) and 9 and 15 months later. The investigators will collect laboratory measures of nutrition status, anthropometrics, Dual-Energy X-Ray Absorptiometry (DXA) to measure body composition and bone mineral density (BMD), and indirect calorimetry to measure resting energy expenditure (REE). In addition, three-day food records will be obtained to assess protein intake and overall diet quality.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'This study will recruit adults with PKU who are starting therapy with pegvaliase and currently following a protein-restricted diet with or without medical food.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of phenylketonuria\n* Aged 18-65 years\n* Following a protein-restricted diet with or without medical food within the last 30 days\n* Routine natural protein intake from food is less than the RDA for protein based on weight (0.8 g/kg/day).\n* If not consuming animal proteins, recommended protein intake is 115% RDA due to lower bioavailability of plant proteins.\n* Within ±90 days of starting treatment with pegvaliase at time of study enrollment\n\nExclusion Criteria:\n\n* Unable to consent to study\n* Under age 18 or over age 65 years\n* Routine natural protein intake is greater than the RDA for protein (0.8 g/kg/d) within the last 30 days.\n* Females who are currently pregnant or lactating'}, 'identificationModule': {'nctId': 'NCT03856203', 'briefTitle': 'Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase', 'organization': {'class': 'OTHER', 'fullName': "Boston Children's Hospital"}, 'officialTitle': 'Longitudinal Assessment of Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase', 'orgStudyIdInfo': {'id': 'IRB-P00029889'}}, 'contactsLocationsModule': {'locations': [{'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': "Boston Children's Hospital", 'class': 'OTHER'}, 'collaborators': [{'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Principal Investigator', 'investigatorFullName': 'Krista Viau', 'investigatorAffiliation': "Boston Children's Hospital"}}}, 'annotationSection': {'annotationModule': {'unpostedAnnotation': {'unpostedEvents': [{'date': '2024-06-17', 'type': 'RELEASE'}, {'date': '2024-09-27', 'type': 'RESET'}], 'unpostedResponsibleParty': "Krista Viau, Principal Investigator, Boston Children's Hospital"}}}}