NCT03775174|Unknown
Expanded Access to Mepsevii
1 other identifier
UX003-EAP
Study Type
expanded_access
Target
N/A
Locations
0 countries
Sites
N/A
Timeline
RegisteredDec 2018
Brief Summary
Individual patient expanded access requests may be considered for patients who have no other treatment options
Trial Health
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 11, 2018
Completed2 days until next milestone
First Posted
Study publicly available on registry
December 13, 2018
CompletedLast Updated
January 12, 2026
Status Verified
January 1, 2026
First QC Date
December 11, 2018
Last Update Submit
January 8, 2026
Conditions
Keywords
Expanded AccessCompassionate Use
Interventions
MepseviiDRUG
Also known as: UX003, recombinant human beta-glucuronidase, rhGUS, vestronidase alfa
Eligibility Criteria
Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Related Links
MeSH Terms
Conditions
Mucopolysaccharidosis VII
Interventions
vestronidase alfa
Condition Hierarchy (Ancestors)
MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases
Central Study Contacts
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 11, 2018
First Posted
December 13, 2018
Last Updated
January 12, 2026
Record last verified: 2026-01