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 groups

Locations

14
Argentina
Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.C1425FNG, Buenos Aires Argentina
Brazil
Hospital de Clínicas de Porto Alegre90035-003, Porto AlegreRio Grande do Sul, Brazil
France
Centre Hospitalier Universitaire La Timone13005, MarseilleProvence-Alpes-Côte d'Azur Region, France
Germany
Universitätsmedizin der Johannes Gutenberg-Universität Mainz55131, Mainz Germany

Sponsors and collaborators

Ultragenyx Pharmaceutical Inc

Lead sponsor