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An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

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

Status
NO_LONGER_AVAILABLE
Phases
Unknown
Study type
Expanded Access
Source
ClinicalTrials.gov
Registry ID
NCT02097251
Enrollment
Unknown
Registered
2014-03-27
Start date
Unknown
Completion date
Unknown
Last updated
2017-02-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Mucopolysaccharidosis Type 7

Keywords

Mucopolysaccharidosis Type 7, Sly syndrome, UX003, Enzyme Replacement Therapy, rare disease, Lysosomal Storage Disease, metabolic disorder

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.

Interventions

DRUGUX003

Open Label

Sponsors

Ultragenyx Pharmaceutical Inc
CollaboratorINDUSTRY
Joyce Fox
Lead SponsorOTHER

Eligibility

Sex/Gender
MALE

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)

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026