Ignite Creation Date:
2025-12-24 @ 10:35 PM
Ignite Modification Date:
2025-12-25 @ 8:06 PM
Study NCT ID:
NCT03604835
Status:
RECRUITING
Last Update Posted:
2025-11-25
First Post:
2018-07-18
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Mucopolysaccharidosis VII Disease Monitoring Program
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Turkey (Türkiye)']}, 'conditionBrowseModule': {'meshes': [{'id': 'D016538', 'term': 'Mucopolysaccharidosis VII'}], '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'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 50}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2018-01-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2033-05', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-21', 'studyFirstSubmitDate': '2018-07-18', 'studyFirstSubmitQcDate': '2018-07-27', 'lastUpdatePostDateStruct': {'date': '2025-11-25', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2018-07-30', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2033-05', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Clinical Course of MPS VII Disease', 'timeFrame': '10 years', 'description': 'To characterize MPS VII disease presentation and progression over time in patients treated and not treated with vestronidase alfa'}, {'measure': 'Long-term Effectiveness of Vestronidase Alfa', 'timeFrame': '10 years', 'description': 'To evaluate longitudinal change in biomarker(s), clinical assessments and patient/caregiver reported outcomes to examine the effectiveness of vestronidase alfa'}, {'measure': 'Long-term Safety of Vestronidase Alfa', 'timeFrame': '10 years', 'description': 'Hypersensitivity reactions, immunogenicity and other safety outcomes will be assessed to examine the long-term safety of vestronidase alfa.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['UX003', 'Mepsevii', 'vestronidase alfa-vjbk', 'vestronidase alfa', 'recombinant human beta-glucuronidase'], 'conditions': ['Mucopolysaccharidosis VII', 'MPS VII', 'MPS 7', 'Sly Syndrome']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://ultrarareadvocacy.com/conditions-we-study/mucopolysaccharidosis-type-vii-mps-vii/', 'label': 'Ultragenyx Patient Advocacy/MPS 7 Disease Information'}, {'url': 'http://www.ultragenyx.com/our-research/our-clinical-trial-transparency-commitment/', 'label': 'Ultragenyx Transparency Commitment'}]}, 'descriptionModule': {'briefSummary': 'The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.', 'detailedDescription': 'The Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP) is a global, prospective, multicenter, longitudinal protocol designed to characterize MPS VII disease presentation and progression, assess long-term effectiveness and safety of vestronidase alfa, including hypersensitivity reactions and immunogenicity , as well as prospectively investigate longitudinal change across biomarker(s), clinical assessments, and patient/ caregiver-reported outcome measures in a representative population. The aim of this DMP is to collect data on patients with MPS VII to provide a comprehensive dataset on the clinical presentation, heterogeneity, and disease progression, and meaningful standardized ICH GCP-quality data collected in-clinic across multiple sites globally. The DMP is not a randomized study and both treated and untreated patients will be enrolled.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients with a confirmed diagnosis of MPS VII, including patients who already received vestronidase alfa in an Ultragenyx clinical trial or early access/ compassionate use program, and patients not receiving vestronidase alfa.\n\nPatients who were previously enrolled in an Ultragenyx-sponsored clinical trial may participate in the DMP if they have completed or discontinued from the clinical trial.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic or mutation analysis.\n* Willing and able to provide written informed consent or, in the case of patients under the age of 18 (or below adult ages as defined by local laws and regulations) or patients \\>18 years of age who have cognitive deficiencies, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the DMP has been explained, and prior to any research-related procedures.\n* Willing to comply with DMP visit schedule.\n\nExclusion Criteria:\n\n* Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.'}, 'identificationModule': {'nctId': 'NCT03604835', 'briefTitle': 'Mucopolysaccharidosis VII Disease Monitoring Program', 'organization': {'class': 'INDUSTRY', 'fullName': 'Ultragenyx Pharmaceutical Inc'}, 'officialTitle': 'Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)', 'orgStudyIdInfo': {'id': 'UX003-CL401'}, 'secondaryIdInfos': [{'id': 'EUPAS25082', 'type': 'OTHER', 'domain': 'EU PAS Number'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'Patients with MPS VII receiving vestronidase-alfa', 'description': 'via prescription, or early access/ compassionate use program', 'interventionNames': ['Other: No Intervention']}, {'label': 'Patients with MPS VII not receiving vestronidase-alfa', 'description': 'no treatment or treatment other than vestronidase alfa', 'interventionNames': ['Other: No Intervention']}], 'interventions': [{'name': 'No Intervention', 'type': 'OTHER', 'description': 'Access to any treatment is through authorized commercial use or available expanded access programs only and not as a part of this DMP.', 'armGroupLabels': ['Patients with MPS VII not receiving vestronidase-alfa', 'Patients with MPS VII receiving vestronidase-alfa']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92868', 'city': 'Orange', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'facility': "Children's Hospital of Orange County", 'geoPoint': {'lat': 33.78779, 'lon': -117.85311}}, {'zip': '20010', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'status': 'RECRUITING', 'country': 'United States', 'facility': "Children's National Health System", 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'status': 'RECRUITING', 'country': 'United States', 'facility': "Ann & Robert H. Lurie Children's Hospital of Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '48109', 'city': 'Ann Arbor', 'state': 'Michigan', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'University of Michigan', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}, {'zip': '10016', 'city': 'New York', 'state': 'New York', 'status': 'TERMINATED', 'country': 'United States', 'facility': 'New York University Langone Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '84112', 'city': 'Salt Lake City', 'state': 'Utah', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Bailey', 'role': 'CONTACT'}], 'facility': 'University of Utah Medical Center', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '98105', 'city': 'Seattle', 'state': 'Washington', 'status': 'RECRUITING', 'country': 'United States', 'facility': "Seattle Children's Hospital", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': 'C1425FNG', 'city': 'Buenos Aires', 'status': 'RECRUITING', 'country': 'Argentina', 'facility': 'Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'zip': '90035-003', 'city': 'Porto Alegre', 'state': 'Rio Grande do Sul', 'status': 'RECRUITING', 'country': 'Brazil', 'facility': 'Hospital de Clínicas de Porto Alegre', 'geoPoint': {'lat': -30.03283, 'lon': -51.23019}}, {'zip': '13005', 'city': 'Marseille', 'state': "Provence-Alpes-Côte d'Azur Region", 'status': 'RECRUITING', 'country': 'France', 'facility': 'Centre Hospitalier Universitaire La Timone', 'geoPoint': {'lat': 43.29695, 'lon': 5.38107}}, {'zip': '55131', 'city': 'Mainz', 'status': 'RECRUITING', 'country': 'Germany', 'facility': 'Universitätsmedizin der Johannes Gutenberg-Universität Mainz', 'geoPoint': {'lat': 49.98185, 'lon': 8.28008}}, {'zip': '3015 CN', 'city': 'Rotterdam', 'state': 'South Holland', 'status': 'TERMINATED', 'country': 'Netherlands', 'facility': 'Erasmus University Medical Center Rotterdam', 'geoPoint': {'lat': 51.9225, 'lon': 4.47917}}, {'zip': '4050-651', 'city': 'Porto', 'status': 'RECRUITING', 'country': 'Portugal', 'facility': 'Centro Hospitalar do Porto', 'geoPoint': {'lat': 41.1485, 'lon': -8.61097}}, {'zip': '41013', 'city': 'Seville', 'status': 'RECRUITING', 'country': 'Spain', 'facility': 'Hospital Universitario Virgen del Rocío Pabellón Infantil', 'geoPoint': {'lat': 37.38283, 'lon': -5.97317}}], 'centralContacts': [{'name': 'Patients Contact: Trial Recruitment', 'role': 'CONTACT', 'email': 'trialrecruitment@ultragenyx.com', 'phone': '1-888-756-8657'}, {'name': 'HCPs Contact: Medical Information', 'role': 'CONTACT', 'email': 'medinfo@ultragenyx.com', 'phone': '1-888-756-8657'}], 'overallOfficials': [{'name': 'Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Ultragenyx Pharmaceuticals Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ultragenyx Pharmaceutical Inc', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}