Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
Ultragenyx Pharmaceutical Inc
Summary
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.
Description
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 dat…
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- No
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. Ex…
Interventions
- OtherNo Intervention
Access to any treatment is through authorized commercial use or available expanded access programs only and not as a part of this DMP.
Locations (14)
- Children's Hospital of Orange CountyOrange, California
- Children's National Health SystemWashington D.C., District of Columbia
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, Illinois
- University of MichiganAnn Arbor, Michigan
- New York University Langone Medical CenterNew York, New York
- University of Utah Medical CenterSalt Lake City, Utah