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

NO_LONGER_AVAILABLE

Conditions studied: Mucopolysaccharidosis Type 7

In brief

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.

Key facts

Study ID

NCT02097251

Run by

Joyce Fox

Last updated by the study team

2017-02-06

Who can join

Age: any. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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)

Where it is running

• Cohen Children's Medical Center — New Hyde Park, New York, United States

Full record on ClinicalTrials.gov

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