Expanded Access to Mepsevii
July 29, 2026 updated by: Ultragenyx Pharmaceutical Inc
Individual patient expanded access requests may be considered for patients who have no other treatment options
Study Overview
Status
Available
Conditions
Intervention / Treatment
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.
Study Type
Expanded Access
Expanded Access Type
- Individual Patients
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
N/A
Description
Inclusion Criteria:
-
Exclusion Criteria:
-
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Registration Dates
First Submitted
December 11, 2018
First Submitted That Met QC Criteria
December 11, 2018
First Posted (Actual)
December 13, 2018
Study Record Updates
Last Update Posted (Actual)
July 30, 2026
Last Update Submitted That Met QC Criteria
July 29, 2026
Last Verified
July 1, 2026
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Metabolism, Inborn Errors
- Genetic Diseases, Inborn
- Metabolic Diseases
- Connective Tissue Diseases
- Carbohydrate Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Mucinoses
- Mucopolysaccharidoses
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities
- Nutritional and Metabolic Diseases
- Skin and Connective Tissue Diseases
- Mucopolysaccharidosis VII
- vestronidase alfa
Other Study ID Numbers
- UX003-EAP
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.