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Recruiting NCT03604835

Mucopolysaccharidosis VII Disease Monitoring Program

Conditions: Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome

Sex: All
Healthy volunteers: No
Enrollment: 50
Sponsor: Ultragenyx Pharmaceutical Inc

Location: Children's Hospital of Orange County Orange California

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 Criteria

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.

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT03604835). StuddyBuddy aggregates publicly available trial information.