Mucopolysaccharidosis VII Disease Monitoring Program
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorUltragenyx Pharmaceutical Inc
About this trial
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
Qualifiers
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.
Disqualifiers
Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.
Trial design
Treatments tested in this trial
- No Intervention
Treatment groups
50 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov