RECRUITINGOBSERVATIONAL
Mucopolysaccharidosis VII Disease Monitoring Program
Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
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.
Who May Be Eligible (Plain English)
Who May Qualify:
- Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic or mutation analysis.
- Willing and able to provide written willing to sign a consent form 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 willing to sign a consent form 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.
Who Should NOT Join This Trial:
- Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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.
Treatments Being Tested
OTHER
No 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)
Children's Hospital of Orange County
Orange, California, United States
Children's National Health System
Washington D.C., District of Columbia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Michigan
Ann Arbor, Michigan, United States
New York University Langone Medical Center
New York, New York, United States
University of Utah Medical Center
Salt Lake City, Utah, United States
Seattle Children's Hospital
Seattle, Washington, United States
Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.
Buenos Aires, Argentina
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil
Centre Hospitalier Universitaire La Timone
Marseille, Provence-Alpes-Côte d'Azur Region, France
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
Mainz, Germany
Erasmus University Medical Center Rotterdam
Rotterdam, South Holland, Netherlands
Centro Hospitalar do Porto
Porto, Portugal
Hospital Universitario Virgen del Rocío Pabellón Infantil
Seville, Spain