An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
NCT ID: NCT02097251
Last Updated: 2017-02-06
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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Brief Summary
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Detailed Description
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Conditions
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Interventions
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UX003
Open Label
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay or genetic testing confirming diagnosis.
* Written informed consent of parent(s) or legal guardian(s)
MALE
No
Sponsors
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Ultragenyx Pharmaceutical Inc
INDUSTRY
Joyce Fox
OTHER
Responsible Party
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Joyce Fox
Chief of Medical Genetics at Cohen Children's Medical Center
Locations
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Cohen Children's Medical Center
New Hyde Park, New York, United States
Countries
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Other Identifiers
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13-606A
Identifier Type: -
Identifier Source: org_study_id
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