Mucopolysaccharidosis VII Clinical Trial
Official title:
Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
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.
Status | Recruiting |
Enrollment | 50 |
Est. completion date | May 2033 |
Est. primary completion date | May 2033 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | Inclusion Criteria: - Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic or mutation analysis. - 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. - Willing to comply with DMP visit schedule. Exclusion Criteria: - Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx. |
Country | Name | City | State |
---|---|---|---|
Argentina | Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L. | Buenos Aires | |
Brazil | Hospital de Clínicas de Porto Alegre | Porto Alegre | Rio Grande Do Sul |
France | Centre Hospitalier Universitaire La Timone | Marseille | Provence Alpes Cote D'Azur |
Germany | Universitätsmedizin der Johannes Gutenberg-Universität Mainz | Mainz | |
Netherlands | Erasmus University Medical Center Rotterdam | Rotterdam | Zuid-Holland |
Portugal | Centro Hospitalar do Porto | Porto | |
Spain | Hospital Universitario Virgen del Rocío Pabellón Infantil | Sevilla | |
Turkey | Gazi University | Ankara | |
United States | University of Michigan | Ann Arbor | Michigan |
United States | Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois |
United States | New York University Langone Medical Center | New York | New York |
United States | Children's Hospital of Orange County | Orange | California |
United States | University of Utah Medical Center | Salt Lake City | Utah |
United States | Seattle Children's Hospital | Seattle | Washington |
United States | Children's National Health System | Washington | District of Columbia |
Lead Sponsor | Collaborator |
---|---|
Ultragenyx Pharmaceutical Inc |
United States, Argentina, Brazil, France, Germany, Netherlands, Portugal, Spain, Turkey,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Clinical Course of MPS VII Disease | To characterize MPS VII disease presentation and progression over time in patients treated and not treated with vestronidase alfa | 10 years | |
Primary | Long-term Effectiveness of Vestronidase Alfa | To evaluate longitudinal change in biomarker(s), clinical assessments and patient/caregiver reported outcomes to examine the effectiveness of vestronidase alfa | 10 years | |
Primary | Long-term Safety of Vestronidase Alfa | Hypersensitivity reactions, immunogenicity and other safety outcomes will be assessed to examine the long-term safety of vestronidase alfa. | 10 years |
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