RecruitingNCT03604835
Mucopolysaccharidosis VII Disease Monitoring Program
Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
Sponsor
Ultragenyx Pharmaceutical Inc
Enrollment
50 participants
Start Date
Jan 29, 2018
Study Type
OBSERVATIONAL
Conditions
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.
Eligibility
Inclusion Criteria3
- 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 Criteria1
- Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.
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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)
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NCT03604835