Ignite Creation Date:
2025-12-24 @ 5:29 PM
Ignite Modification Date:
2025-12-25 @ 3:03 PM
Study NCT ID:
NCT00144768
Status:
COMPLETED
Last Update Posted:
2014-05-05
First Post:
2005-09-02
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D008059', 'term': 'Mucopolysaccharidosis I'}], 'ancestors': [{'id': 'D009083', 'term': 'Mucopolysaccharidoses'}, {'id': 'D002239', 'term': 'Carbohydrate 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': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D017520', 'term': 'Mucinoses'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D007068', 'term': 'Iduronidase'}], 'ancestors': [{'id': 'D006026', 'term': 'Glycoside Hydrolases'}, {'id': 'D006867', 'term': 'Hydrolases'}, {'id': 'D004798', 'term': 'Enzymes'}, {'id': 'D045762', 'term': 'Enzymes and Coenzymes'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 25}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2004-07'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-04', 'completionDateStruct': {'date': '2007-05', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-05-02', 'studyFirstSubmitDate': '2005-09-02', 'studyFirstSubmitQcDate': '2005-09-02', 'lastUpdatePostDateStruct': {'date': '2014-05-05', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2005-09-05', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2007-05', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Urinary GAG (glycosaminoglycans)', 'timeFrame': 'Up to 4 years'}, {'measure': 'Immunogenicity Testing', 'timeFrame': 'Up to 4 years'}], 'secondaryOutcomes': [{'measure': 'Safety', 'timeFrame': 'Up to 4 years'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Mucopolysaccharidosis I', "Hurler's Syndrome", 'Hurler-Scheie Syndrome', "Scheie's Syndrome"]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Have a documented diagnosis of MPS I confirmed by measurable signs and symptoms and deficient α-L-iduronidase activity (\\<10% of the lower limit of normal).\n* For a patients receiving Aldurazyme therapy prior to study entry: Have available as baseline data the results of urinary GAG levels and IgG antibody titers collected prior to the patient's first Aldurazyme infusion.\n* Provide signed, written informed consent prior to any protocol-related procedures being performed. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years.\n\nExclusion Criteria:\n\n* Have previously received Aldurazyme without the collection of baseline samples as specified.\n* Have a suspected hypersensitivity to Aldurazyme or a know hypersensitivity to components of infusion solution.\n* Have received a Hematopoietic Stem Cell Transplantation, injection fibroblast therapy, or major organ transplant.\n* Are receiving chronic immunosuppressant therapy.\n* Have a medical condition, serious intercurrent illness, or other extenuating circumstances that may interfere with study compliance including all prescribed evaluations and follow-up activities.\n* Are pregnant or lactating\n* Have received investigational drug within 30 days prior to study enrollment"}, 'identificationModule': {'nctId': 'NCT00144768', 'briefTitle': 'A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sanofi'}, 'officialTitle': 'A Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients With Mucopolysaccharidosis I (MPS I) Being Treated With Aldurazyme® (Laronidase).', 'orgStudyIdInfo': {'id': 'ALID02003'}}, 'armsInterventionsModule': {'interventions': [{'name': 'laronidase', 'type': 'DRUG', 'otherNames': ['Recombinant Human Alpha-L-Iduronidase, Aldurazyme®'], 'description': 'dose of 0.58mg/kg body weight IV every week'}]}, 'contactsLocationsModule': {'locations': [{'zip': '90027', 'city': 'Los Angles', 'state': 'California', 'country': 'United States', 'facility': 'Childrens Hospital Los Angeles'}, {'zip': '06106', 'city': 'Hartford', 'state': 'Connecticut', 'country': 'United States', 'facility': "Connecticut Children's Medical Center", 'geoPoint': {'lat': 41.76371, 'lon': -72.68509}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': "Children's Hospital of Wisconsin", 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}], 'overallOfficials': [{'name': 'Medical Monitor', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Genzyme, a Sanofi Company'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Genzyme, a Sanofi Company', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'BioMarin/Genzyme LLC', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}