Ignite Creation Date:
2025-12-25 @ 3:44 AM
Ignite Modification Date:
2025-12-26 @ 2:30 AM
Study NCT ID:
NCT05017402
Status:
NOT_YET_RECRUITING
Last Update Posted:
2021-08-23
First Post:
2021-08-05
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Higher Dose of Alglucosidase Alpha for Pompe Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006009', 'term': 'Glycogen Storage Disease Type II'}], 'ancestors': [{'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'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': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D006008', 'term': 'Glycogen Storage Disease'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C509951', 'term': 'GAA protein, human'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'OTHER', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 36}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2021-09-01', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-08', 'completionDateStruct': {'date': '2026-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2021-08-17', 'studyFirstSubmitDate': '2021-08-05', 'studyFirstSubmitQcDate': '2021-08-17', 'lastUpdatePostDateStruct': {'date': '2021-08-23', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-08-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-12-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Forced vital capacity', 'timeFrame': 'For patient older than 3-year-old, first test before study, then every six months, up to 2-years.', 'description': 'Pulmonary function test (Units: percentage of predictive value)'}, {'measure': 'Peak expiratory flow', 'timeFrame': 'For patient older than 3-year-old, first test before study, then every six months, up to 2-years.', 'description': 'Pulmonary function test (Units: percentage of predictive value)'}, {'measure': 'Polysomnography', 'timeFrame': 'For patient older than 6-month-old, first test before study, then every six months, up to 2-years.', 'description': 'Comprehensive test used to diagnose sleep disorders.'}], 'secondaryOutcomes': [{'measure': 'uGLC4', 'timeFrame': 'uGLC4 will be tested before the treatment, then every three months, up to 2-years.', 'description': 'Urine glucose tetrasaccharide (Units: mmol/mol Creatinine).'}, {'measure': 'CK', 'timeFrame': 'CK will be tested before the treatment, then every three months, up to 2-years.', 'description': 'Blood creatine kinase (Units: units per liter, U/L).'}, {'measure': 'AST', 'timeFrame': 'AST will be tested before the treatment, then every three months, up to 2-years.', 'description': 'Blood aspartate aminotransferase (Units: units per liter, U/L).'}, {'measure': 'ALT', 'timeFrame': 'ALT will be tested before the treatment, then every three months, up to 2-years.', 'description': 'Blood alanine aminotransferase (Units: units per liter, U/L).'}, {'measure': 'Body weight', 'timeFrame': 'The body weight will be monitored before the treatment, then every two weeks, up to 2-years.', 'description': 'Change of body weight (Unit: kilogram, kg)'}, {'measure': 'Body height', 'timeFrame': 'The body height will be monitored before the treatment, then every two weeks, up to 2-years.', 'description': 'Change of body height (Units: centimeter, cm)'}, {'measure': 'Antibody titers', 'timeFrame': 'First test will be one month later after first ERT, then every six months, up to 2-years.', 'description': 'Alglucosidase alfa IgG antibody titer'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Glycogen Storage Disease Type II']}, 'descriptionModule': {'briefSummary': 'This study is aimed to investigate that whether the higher dose ERT improve safety and clinical outcomes of Pompe disease patients. Also, wish to develop a new therapeutic recommendation and hope that it could improve the long-term outcomes of Pompe diesease patients.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '60 Years', 'minimumAge': '0 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'The observational population included patients of Pompe disease who were diagnosed and treated in our series.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Diagnosis of Pompe disease\n2. Currently receiving ERT with regular clinic visits\n3. Agree to sign informed consent\n\nExclusion Criteria:\n\n1. Lost follow-up for clinical visits\n2. Allergy to Myozyme\n3. Refuse to sign informed consent'}, 'identificationModule': {'nctId': 'NCT05017402', 'briefTitle': 'Higher Dose of Alglucosidase Alpha for Pompe Disease', 'organization': {'class': 'OTHER_GOV', 'fullName': 'Taipei Veterans General Hospital, Taiwan'}, 'officialTitle': 'Higher Dose of Alglucosidase Alpha for Pompe Disease: Long-term Follow-up Study', 'orgStudyIdInfo': {'id': '2021-02-019A'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Alglucosidase Alfa', 'type': 'DRUG', 'description': 'Standard dose: 20-25 mg/kg every other week; Higher dose: \\>25 mg/kg every other week.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Taipei Veterans General Hospital, Taiwan', 'class': 'OTHER_GOV'}, 'responsibleParty': {'type': 'SPONSOR'}}}}