UX003: Clinical Trials

4 completed, 1 available, 1 no longer available. 2 Phase 2, 2 Phase 3, 1 Phase 1.

By phase and status

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) UX003 - Sly Syndrome, MPS VII	Phase 3	Completed	Ultragenyx Pharmaceutical Inc	12	2015-11-10	2020-07-30
Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age UX003 - Sly Syndrome, MPS VII	Phase 2	Completed	Ultragenyx Pharmaceutical Inc	8	2015-07-21	2019-10-30
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) UX003, Placebo - MPS 7, Sly Syndrome	Phase 3	Completed	Ultragenyx Pharmaceutical Inc	12	2014-12-01	2020-07-30
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) UX003 - Mucopolysaccharidosis Type 7	Phase 1 / Phase 2	Completed	Ultragenyx Pharmaceutical Inc	3	2013-11-01	2019-01-04
Expanded Access to Mepsevii Mepsevii - MPS VII, Mucopolysaccharidosis VII		Available	Ultragenyx Pharmaceutical Inc			2026-07-30
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient UX003 - Mucopolysaccharidosis Type 7		No longer available	Joyce Fox			2017-02-06