Ignite Creation Date:
2025-12-25 @ 5:05 AM
Ignite Modification Date:
2026-03-05 @ 10:46 PM
Study NCT ID:
NCT01856218
Status:
COMPLETED
Last Update Posted:
2019-01-04
First Post:
2013-05-08
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
Sponsor:
Organization:
Study Design
Study Type:
INTERVENTIONAL
Expanded Access Type Individual:
None
Expanded Access Type Intermediate:
None
Expanded Access Type Treatment:
None
Patient Registry:
None
Target Duration:
None
Design Allocation:
Design Intervention Model:
Design Intervention Model Description:
Design Masking Description:
Bio Spec Retention:
None
Bio Spec Description:
None
Enrollment Count:
Enrollment Type:
Design Primary Purpose:
Design Masking:
Time Perspective List:
Who Masked List: