Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
50 participants
OBSERVATIONAL
2018-01-29
2033-05-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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OTHER
PROSPECTIVE
Study Groups
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Patients with MPS VII receiving vestronidase-alfa
via prescription, or early access/ compassionate use program
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.
Patients with MPS VII not receiving vestronidase-alfa
no treatment or treatment other than vestronidase alfa
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.
Interventions
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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.
Eligibility Criteria
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Inclusion Criteria
* 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
ALL
No
Sponsors
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Ultragenyx Pharmaceutical Inc
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Ultragenyx Pharmaceuticals Inc.
Locations
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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, 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, Sevilla, Spain
Countries
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Central Contacts
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Related Links
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Ultragenyx Patient Advocacy/MPS 7 Disease Information
Ultragenyx Transparency Commitment
Other Identifiers
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EUPAS25082
Identifier Type: OTHER
Identifier Source: secondary_id
UX003-CL401
Identifier Type: -
Identifier Source: org_study_id
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