Back to resultsAn Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Primary Purpose
Mucopolysaccharidosis Type 7
Status
No longer available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
UX003
Sponsored by
About this trial
This is an expanded access trial for Mucopolysaccharidosis Type 7 focused on measuring Mucopolysaccharidosis Type 7, Sly syndrome, UX003, Enzyme Replacement Therapy, rare disease, Lysosomal Storage Disease, metabolic disorder
Eligibility Criteria
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)
Sites / Locations
- Cohen Children's Medical Center
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT02097251
First Posted
March 24, 2014
Last Updated
February 2, 2017
Sponsor
Joyce Fox
Collaborators
Ultragenyx Pharmaceutical Inc
1. Study Identification
Unique Protocol Identification Number
NCT02097251
Brief Title
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Official Title
An Open-Label Treatment Protocol With UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy for an Advanced Stage Mucopolysaccharidosis Type 7 (MPS 7) Patient
Study Type
Expanded Access
2. Study Status
Record Verification Date
February 2017
Overall Recruitment Status
No longer available
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Joyce Fox
Collaborators
Ultragenyx Pharmaceutical Inc
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis Type 7
Keywords
Mucopolysaccharidosis Type 7, Sly syndrome, UX003, Enzyme Replacement Therapy, rare disease, Lysosomal Storage Disease, metabolic disorder
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
UX003
Other Intervention Name(s)
recombinant human beta glucuronidase, rhGUS
Intervention Description
Open Label
10. Eligibility
Sex
Male
Eligibility Criteria
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)
Facility Information:
Facility Name
Cohen Children's Medical Center
City
New Hyde Park
State/Province
New York
ZIP/Postal Code
11040
Country
United States
12. IPD Sharing Statement
Learn more about this trial
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
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