Ignite Creation Date:
2025-12-24 @ 9:24 PM
Ignite Modification Date:
2025-12-25 @ 7:10 PM
Study NCT ID:
NCT00763932
Status:
COMPLETED
Last Update Posted:
2014-02-05
First Post:
2008-09-30
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies
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': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 7}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2003-04'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-02', 'completionDateStruct': {'date': '2006-07', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-02-04', 'studyFirstSubmitDate': '2008-09-30', 'studyFirstSubmitQcDate': '2008-09-30', 'lastUpdatePostDateStruct': {'date': '2014-02-05', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2008-10-01', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2006-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The objective of this extension study was to monitor the long-term safety and efficacy', 'timeFrame': '3 years'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Pompe Disease Infantile-Onset', 'Glycogen Storage Disease Type II']}, 'descriptionModule': {'briefSummary': 'This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* The patient was enrolled in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502\n* The patient's legal guardian(s) provided written informed consent prior to any study related procedures being performed\n* The patient and his/her guardian(s) were able to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.\n\nExclusion Criteria:\n\n* Patients were excluded from this study if they did not meet the specific inclusion criteria, or if the patient experienced any unmanageable AE in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502 (as determined and agreed upon by the Principal Investigator and Genzyme Corporation), due to Synpac rhGAA therapy, that would preclude continuing therapy"}, 'identificationModule': {'nctId': 'NCT00763932', 'briefTitle': 'Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sanofi'}, 'officialTitle': 'A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies', 'orgStudyIdInfo': {'id': 'AGLU02003'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '1', 'interventionNames': ['Biological: Myozyme']}], 'interventions': [{'name': 'Myozyme', 'type': 'BIOLOGICAL', 'otherNames': ['Alglucosidase alfa'], 'description': '10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow', 'armGroupLabels': ['1']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital of Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'city': 'Orange', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital of Orange Country", 'geoPoint': {'lat': 33.78779, 'lon': -117.85311}}, {'city': 'Omaha', 'state': 'Nebraska', 'country': 'United States', 'facility': 'University of Nebraska Medical Center', 'geoPoint': {'lat': 41.25626, 'lon': -95.94043}}, {'city': 'New Brunswick', 'state': 'New Jersey', 'country': 'United States', 'facility': "Institute for Genetic Medicine, Saint Peter's University Hospital", 'geoPoint': {'lat': 40.48622, 'lon': -74.45182}}, {'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'New York University [NYU] School of Medicine', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'city': 'Lyon', 'country': 'France', 'facility': 'Pediatrique Hospital Debrousse', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'city': 'Orléans', 'country': 'France', 'facility': 'Hôpital Porte Madeleine', 'geoPoint': {'lat': 47.90248, 'lon': 1.90407}}, {'city': 'Johannesburg', 'country': 'South Africa', 'facility': 'The Morningside Clinic', 'geoPoint': {'lat': -26.20227, 'lon': 28.04363}}], 'overallOfficials': [{'name': 'Medical Monitor', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Genzyme, a Sanofi Company'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Genzyme, a Sanofi Company', 'class': 'INDUSTRY'}, 'responsibleParty': {'oldNameTitle': 'Medical Monitor', 'oldOrganization': 'Genzyme Corporation'}}}}