Expanded Access to Mepsevii
Primary Purpose
MPS VII, Mucopolysaccharidosis VII, Sly Syndrome
Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
Mepsevii
Sponsored by
About this trial
This is an expanded access trial for MPS VII focused on measuring Expanded Access, Compassionate Use
Eligibility Criteria
Inclusion Criteria:
-
Exclusion Criteria:
-
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT03775174
First Posted
December 11, 2018
Last Updated
September 1, 2023
Sponsor
Ultragenyx Pharmaceutical Inc
1. Study Identification
Unique Protocol Identification Number
NCT03775174
Brief Title
Expanded Access to Mepsevii
Study Type
Expanded Access
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
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
Name of the Sponsor
Ultragenyx Pharmaceutical Inc
4. Oversight
5. Study Description
Brief Summary
Individual patient expanded access requests may be considered for patients who have no other treatment options
Detailed Description
Expanded access may be available outside of the US in countries prior to approval by the local regulatory agencies. For full details, please visit the links provided below.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
MPS VII, Mucopolysaccharidosis VII, Sly Syndrome
Keywords
Expanded Access, Compassionate Use
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Mepsevii
Other Intervention Name(s)
UX003, recombinant human beta-glucuronidase, rhGUS, vestronidase alfa
10. Eligibility
Sex
All
Eligibility Criteria
Inclusion Criteria:
-
Exclusion Criteria:
-
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Early Access
Phone
1-415-483-8800
Email
EarlyAccess@ultragenyx.com
12. IPD Sharing Statement
Links:
URL
https://www.ultragenyx.com/our-purpose/supporting-access-for-patients/?#investigational-therapies
Description
Access to Investigational Therapies / Requesting Access
URL
https://www.mepsevii.com/en/
Description
Mepsevii Patient and Prescribing Information Page
Learn more about this trial
Expanded Access to Mepsevii
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