Ignite Creation Date:
2025-12-26 @ 12:14 PM
Ignite Modification Date:
2025-12-31 @ 2:32 PM
Study NCT ID:
NCT00299000
Status:
COMPLETED
Last Update Posted:
2011-07-22
First Post:
2006-03-02
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009087', 'term': 'Mucopolysaccharidosis VI'}], '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': 'C508864', 'term': 'galsulfase'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@bmrn.com', 'phone': '800-983-4587', 'title': 'Medical Information Services', 'organization': 'BioMarin Pharmaceutical Inc.'}, 'certainAgreement': {'otherDetails': 'The first publication of the results shall be made by the Sponsor in a joint publication. If such a multicenter publication is not submitted within 12 months after conclusion of the study, the PI may publish the results from site individually, subject however to comply with the other terms of the agreement.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'Given the small number of patients (4) represented in this study, the outcomes observed in this study may not reflect or predict outcomes observed by physicians in clinical practice.'}}, 'adverseEventsModule': {'timeFrame': 'All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.', 'eventGroups': [{'id': 'EG000', 'title': 'Naglazyme, 1.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks', 'otherNumAtRisk': 2, 'otherNumAffected': 2, 'seriousNumAtRisk': 2, 'seriousNumAffected': 2}, {'id': 'EG001', 'title': 'Naglazyme, 2.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks', 'otherNumAtRisk': 2, 'otherNumAffected': 2, 'seriousNumAtRisk': 2, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 4, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 15, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 8, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 31, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Umbilical hernia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Upper respiratory tract infections', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 9, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}], 'seriousEvents': [{'term': 'Adenoidal hypertrophy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Cast application', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Febrile convulsion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Poor venous access', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Respiratory distress', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Scoliosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Umbilical hernia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'SECONDARY', 'title': 'Change in Urinary Glycosaminoglycan Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Naglazyme, 1.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks'}, {'id': 'OG001', 'title': 'Naglazyme, 2.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks'}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '1041.87', 'spread': '86.32', 'groupId': 'OG000'}, {'value': '698.95', 'spread': '142.34', 'groupId': 'OG001'}]}]}, {'title': 'Week 52', 'categories': [{'measurements': [{'value': '261.15', 'spread': '54.38', 'groupId': 'OG000'}, {'value': '178.10', 'spread': '42.43', 'groupId': 'OG001'}]}]}, {'title': 'Change from Baseline to Week 52', 'categories': [{'measurements': [{'value': '-780.72', 'spread': '140.69', 'groupId': 'OG000'}, {'value': '-520.85', 'spread': '99.91', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'minimum 52 weeks of dosing', 'description': 'Change in urinary GAG levels was calculated from baseline to week 52 of treatment.', 'unitOfMeasure': 'ug/mg creatinine', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Intention to treat.'}, {'type': 'PRIMARY', 'title': 'Change in Height', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Naglazyme, 1.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks'}, {'id': 'OG001', 'title': 'Naglazyme, 2.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks'}], 'classes': [{'title': 'Height at baseline', 'categories': [{'measurements': [{'value': '67.3', 'spread': '5.1', 'groupId': 'OG000'}, {'value': '80.3', 'spread': '1.8', 'groupId': 'OG001'}]}]}, {'title': 'Height at week 52', 'categories': [{'measurements': [{'value': '81.9', 'spread': '1.8', 'groupId': 'OG000'}, {'value': '91.3', 'spread': '5.2', 'groupId': 'OG001'}]}]}, {'title': 'Change in height', 'categories': [{'measurements': [{'value': '14.6', 'spread': '6.9', 'groupId': 'OG000'}, {'value': '11.0', 'spread': '7.0', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '52 weeks', 'unitOfMeasure': 'centimeters', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Intention to treat.'}, {'type': 'PRIMARY', 'title': 'Change in Weight', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Naglazyme, 1.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks'}, {'id': 'OG001', 'title': 'Naglazyme, 2.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks'}], 'classes': [{'title': 'weight at baseline', 'categories': [{'measurements': [{'value': '7.1', 'spread': '.3', 'groupId': 'OG000'}, {'value': '10.2', 'spread': '.6', 'groupId': 'OG001'}]}]}, {'title': 'weight at week 52', 'categories': [{'measurements': [{'value': '11.3', 'spread': '.3', 'groupId': 'OG000'}, {'value': '13.3', 'spread': '1.0', 'groupId': 'OG001'}]}]}, {'title': 'change in weight', 'categories': [{'measurements': [{'value': '4.2', 'spread': '.6', 'groupId': 'OG000'}, {'value': '3.1', 'spread': '1.6', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '52 weeks', 'unitOfMeasure': 'kilograms', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Change in Haed Circumference', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Naglazyme, 1.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks'}, {'id': 'OG001', 'title': 'Naglazyme, 2.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks'}], 'classes': [{'title': 'head circumference at baseline', 'categories': [{'measurements': [{'value': '43.5', 'spread': '3.0', 'groupId': 'OG000'}, {'value': '49.1', 'spread': '.5', 'groupId': 'OG001'}]}]}, {'title': 'head circumference at 52 weeks', 'categories': [{'measurements': [{'value': '48.5', 'spread': '1.0', 'groupId': 'OG000'}, {'value': '51.8', 'spread': '.8', 'groupId': 'OG001'}]}]}, {'title': 'Change in head circumference', 'categories': [{'measurements': [{'value': '5.0', 'spread': '4.0', 'groupId': 'OG000'}, {'value': '2.7', 'spread': '1.3', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '52 weeks', 'unitOfMeasure': 'centimeter', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Naglazyme, 1.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks'}, {'id': 'FG001', 'title': 'Naglazyme, 2.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Global study sites were hospitals.\n\nFirst Enrollment: 08MAY2006\n\nLast Dose: 30APR2009'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Naglazyme, 1.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks'}, {'id': 'BG001', 'title': 'Naglazyme, 2.0 mg/kg', 'description': 'weekly infusions for minimum of 52 weeks'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age Continuous', 'classes': [{'categories': [{'measurements': [{'value': '6.05', 'spread': '3.89', 'groupId': 'BG000'}, {'value': '12.4', 'spread': '0.42', 'groupId': 'BG001'}, {'value': '9.23', 'spread': '4.31', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'months', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White, non-Hispanic', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'Hispanic/Latino', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Other', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'France', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Portugal', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'PREVENTION', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 4}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2006-05'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2011-07', 'completionDateStruct': {'date': '2009-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2011-07-19', 'studyFirstSubmitDate': '2006-03-02', 'resultsFirstSubmitDate': '2010-05-07', 'studyFirstSubmitQcDate': '2006-03-02', 'lastUpdatePostDateStruct': {'date': '2011-07-22', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2010-09-28', 'studyFirstPostDateStruct': {'date': '2006-03-06', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2010-10-15', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2009-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in Height', 'timeFrame': '52 weeks'}, {'measure': 'Change in Weight', 'timeFrame': '52 weeks'}, {'measure': 'Change in Haed Circumference', 'timeFrame': '52 weeks'}], 'secondaryOutcomes': [{'measure': 'Change in Urinary Glycosaminoglycan Levels', 'timeFrame': 'minimum 52 weeks of dosing', 'description': 'Change in urinary GAG levels was calculated from baseline to week 52 of treatment.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Mucopolysaccharidosis VI', 'Maroteaux-Lamy Syndrome']}, 'referencesModule': {'references': [{'pmid': '33775523', 'type': 'DERIVED', 'citation': 'Garcia P, Phillips D, Johnson J, Martin K, Randolph LM, Rosenfeld H, Harmatz P. Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy. Mol Genet Metab. 2021 May;133(1):100-108. doi: 10.1016/j.ymgme.2021.03.006. Epub 2021 Mar 14.'}, {'pmid': '24108527', 'type': 'DERIVED', 'citation': 'Harmatz PR, Garcia P, Guffon N, Randolph LM, Shediac R, Braunlin E, Lachman RS, Decker C. Galsulfase (Naglazyme(R)) therapy in infants with mucopolysaccharidosis VI. J Inherit Metab Dis. 2014 Mar;37(2):277-87. doi: 10.1007/s10545-013-9654-7. Epub 2013 Oct 10.'}], 'seeAlsoLinks': [{'url': 'http://www.BRMN.com', 'label': 'BioMarin Pharmaceutical Inc Website'}]}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to evaluate the safety and efficacy of two dose levels of Naglazyme in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.', 'detailedDescription': 'The primary objective of the study was to evaluate the efficacy of two dose levels of Naglazyme in preventing the progression of skeletal dysplasia in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.\n\nThe secondary objective of the study was to evaluate the efficacy of the two dose levels of Naglazyme in preventing several measures of disease progression in infants under the age of one year who have MPS VI by monitoring urinary GAGs, gross and fine motor function, cardiac function, vision, hearing, and use of health resources.\n\nThe safety objective of the study was to evaluate the safety of two dose levels of Naglazyme in infants under the age of one year who have MPS VI.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '1 Year', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Signed informed consent by a parent or legal guardian\n* Parent or legal guardian willing and able to comply with all study procedures\n* Equal to or greater than 36 weeks estimated gestational age by physical exam at birth\n* Has a diagnosis of MPS VI based on a documented prenatal diagnosis or fibroblast or leukocyte N-acetylgalactosamine 4-sulfatase (ASB) enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory\n* Is less than one year of age\n* Has no evidence of skeletal dysplasia based on physical exam\n\nExclusion Criteria:\n\n* Parent of legal guardian perceived to be unreliable or unavailable for study participation\n* Use of any investigational drug within 30 days prior to screening, or requirement for any investigational agent prior to completion of all scheduled study assessments\n* Has concurrent disease or condition that would interfere with study participation or safety (i.e., has previously undergone hematopoietic stem cell transplantation such as bone marrow or cord blood transplantation, or major organ transplantation)\n* Any condition that, in the view of the principle investigator, renders the subject at high risk from treatment compliance and/or completing the study\n* Has known hypersensitivity to Naglazyme\n* Has previously received Naglazyme'}, 'identificationModule': {'nctId': 'NCT00299000', 'briefTitle': 'A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioMarin Pharmaceutical'}, 'officialTitle': 'A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With Maroteaux-Lamy Syndrome (MPS VI)', 'orgStudyIdInfo': {'id': 'ASB-008'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'Naglazyme, 1.0 mg/kg', 'description': 'Dose comparison', 'interventionNames': ['Drug: Naglazyme']}, {'type': 'OTHER', 'label': 'Naglazyme, 2.0 mg/kg', 'description': 'Dose Comparison', 'interventionNames': ['Drug: Naglazyme']}], 'interventions': [{'name': 'Naglazyme', 'type': 'DRUG', 'otherNames': ['recombinant human N-acetylgalactosamine 4-sulfatase', 'rh-arylsulfatase B', 'rhASB'], 'description': 'Weekly infusion for minimum of 52 weeks. Naglazyme is diluted in sterile 0.9% sodium chloride solution', 'armGroupLabels': ['Naglazyme, 1.0 mg/kg', 'Naglazyme, 2.0 mg/kg']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital Los Angeles", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '69677', 'city': 'Lyon', 'country': 'France', 'facility': 'Hospital Femme Mere Enfant Centre', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'zip': '3000-076', 'city': 'Coimbra', 'country': 'Portugal', 'facility': 'Hospital PediAtrico de Coimbra', 'geoPoint': {'lat': 40.20686, 'lon': -8.41996}}], 'overallOfficials': [{'name': 'Celeste Decker, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'BioMarin Pharmaceutical'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}, 'responsibleParty': {'oldNameTitle': 'Celeste Decker, MD', 'oldOrganization': 'BioMarin Pharmaceutical Inc.'}}}}