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โ† Trials
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Mucopolysaccharidosis VII Disease Monitoring Program

Recruitingn/aAges any - anyAllUltragenyx Pharmaceutical Inc
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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.
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What it tests

  • No Intervention
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Where

14 sites
Children's Hospital of Orange County
Orange, United States
Children's National Health System
Washington D.C., United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States
University of Michigan
Ann Arbor, United States
New York University Langone Medical Center
New York, United States
University of Utah Medical Center
Salt Lake City, United States
Seattle Children's Hospital
Seattle, United States
Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.
Buenos Aires, Argentina

โ€ฆand 6 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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