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

Mucopolysaccharidosis Type 7 Clinical Trial

Official title:

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

Clinical Trial Details — Status: No longer available

Administrative data

• NCT number: NCT02097251
• Other study ID #: 13-606A
• Status: No longer available
• Phase: N/A
• First received: March 24, 2014
• Last updated: February 2, 2017

Study information

• Verified date: February 2017
• Source: Northwell Health
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

Recruitment information / eligibility

• Status: No longer available
• Enrollment: 0
• Gender: Male
• Age group: N/A and older

Eligibility

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)

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis Type 7

Intervention

Drug:
• UX003
Open Label

Locations

Country	Name	City	State
United States	Cohen Children's Medical Center	New Hyde Park	New York

Sponsors (2)

Lead Sponsor	Collaborator
Joyce Fox	Ultragenyx Pharmaceutical Inc

Country where clinical trial is conducted

United States,