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)
PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment
3
Participants
Timeline
Start Date
November 30, 2013
Primary Completion Date
July 31, 2016
Study Completion Date
July 31, 2016
Conditions
Mucopolysaccharidosis Type 7
Interventions
DRUG
UX003
Trial Locations (1)
Unknown
Manchester Academic Health Science Centre, Manchester
All Listed Sponsors
lead
Ultragenyx Pharmaceutical Inc
INDUSTRY
NCT01856218 - 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) | Biotech Hunter | Biotech Hunter