Ignite Creation Date:
2025-12-24 @ 11:42 PM
Ignite Modification Date:
2026-02-22 @ 4:13 PM
Study NCT ID:
NCT03792451
Status:
COMPLETED
Last Update Posted:
2021-03-04
First Post:
2018-12-31
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Nutrition Status of Adults Treated With Pegvaliase
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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000629004', 'term': 'pegvaliase'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'CROSS_SECTIONAL', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 18}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-01-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-03', 'completionDateStruct': {'date': '2020-12-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-03-02', 'studyFirstSubmitDate': '2018-12-31', 'studyFirstSubmitQcDate': '2019-01-02', 'lastUpdatePostDateStruct': {'date': '2021-03-04', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-01-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2020-12-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Mean concentrations of serum marker(s) of protein status', 'timeFrame': 'through study completion, an average of 1 year'}, {'measure': 'Mean concentrations of serum marker(s) of micronutrient status', 'timeFrame': 'through study completion, an average of 1 year'}, {'measure': 'Mean concentrations of serum marker(s) of essential fatty acid status', 'timeFrame': 'through study completion, an average of 1 year'}, {'measure': 'Mean concentrations of serum marker(s) of cardiovascular status', 'timeFrame': 'through study completion, an average of 1 year'}, {'measure': 'Mean protein intake (g/day) as reported on a three-day food record', 'timeFrame': 'through study completion, an average of 1 year'}, {'measure': 'Mean micronutrient intake (%DRI) as reported on a three-day food record', 'timeFrame': 'through study completion, an average of 1 year'}], 'secondaryOutcomes': [{'measure': 'Mean Healthy Eating Index-2015 scores calculated from the Diet History Questionnaire III', 'timeFrame': 'through study completion, an average of 1 year', 'description': '"The Healthy Eating Index-2015 (HEI-2015) is… used to assess how well a set of foods aligns with the 2015-2020 Dietary Guidelines for Americans. The HEI-2015 includes 13 components that can be summed to a maximum total score of 100 points. The components capture… adequacy components and… moderation components. For the adequacy components, higher scores reflect higher intakes that meet or exceed the standards. For the moderation components, higher scores reflect lower intakes because lower intakes are more desirable. A higher total score indicates a diet that aligns better with the Dietary Guidelines." - https://www.cnpp.usda.gov/sites/default/files/healthy\\_eating\\_index/HEI2015-ScoresForAmericans.pdf\n\nAdequacy components include total fruits, whole fruits, total vegetables, greens and beans, whole grains, dairy, total protein foods, seafood and plant proteins, fatty acids. Moderation components include refined grains, sodium, added sugars, and saturated fats.'}, {'measure': 'Correlation between serum micronutrient concentrations and average micronutrient intake', 'timeFrame': 'through study completion, an average of 1 year'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Phenylketonurias']}, 'descriptionModule': {'briefSummary': 'Conduct a cross-sectional analysis of the nutritional status of individuals with PKU treated with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more months while on pegvaliase treatment. We will evaluate laboratory markers of nutritional status, including markers of protein and micronutrient nutriture. In addition, a three-day food record and food frequency questionnaire 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 cross-sectional study will recruit adults with PKU who have been treated with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more months while on pegvaliase treatment. This may include individuals who participated in the phase III clinical trials for pegvaliase.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Diagnosis of phenylketonuria\n2. Aged 18-65 years\n3. Currently treated with pegvaliase\n4. Have either 1) received treatment with pegvaliase for one or more years or 2) followed an unrestricted diet for 6 or more months while on pegvaliase\n5. Currently following an unrestricted diet that meets or exceeds the RDA for protein (0.8 g/kg/day)\n\nExclusion Criteria:\n\n1. Unable to consent to study\n2. Under age 18 years or over age 65\n3. Routine food intake provides less than the RDA for protein (0.8 g/kg/d)\n4. Consuming any PKU medical foods'}, 'identificationModule': {'nctId': 'NCT03792451', 'briefTitle': 'Nutrition Status of Adults Treated With Pegvaliase', 'organization': {'class': 'OTHER', 'fullName': "Boston Children's Hospital"}, 'officialTitle': 'Nutrition Status of Adults With Phenylketonuria Treated With Pegvaliase', 'orgStudyIdInfo': {'id': 'P00029611'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Pegvaliase', 'type': 'DRUG', 'otherNames': ['Palynziq'], 'description': 'Treated with pegvaliase for one or more years or followed an unrestricted diet for 6 or more months while being treated with pegvaliase.'}]}, 'contactsLocationsModule': {'locations': [{'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}], 'overallOfficials': [{'name': 'Krista Viau, PhD, RD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Boston Children's Hospital"}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': "Boston Children's Hospital", 'class': 'OTHER'}, 'collaborators': [{'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Clinical Nutrition Specialist II', 'investigatorFullName': 'Krista Viau', 'investigatorAffiliation': "Boston Children's Hospital"}}}}