An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
- Conditions
- Mucopolysaccharidosis Type 7
- Registration Number
- NCT02097251
- Lead Sponsor
- Joyce Fox
- Brief Summary
Emergency access granted to treat a single patient with advanced Mucopolysaccharidosis Type 7 with UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy via IV administration every other week (QOW) for up to 144 weeks.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NO_LONGER_AVAILABLE
- Sex
- Male
- Target Recruitment
- Not specified
Inclusion Criteria
- 1 specific patient
- 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)
Exclusion Criteria
Not provided
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Cohen Children's Medical Center
🇺🇸New Hyde Park, New York, United States