NCT02230566View on ClinicalTrials.gov

A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

PHASE3CompletedINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

December 31, 2014

Primary Completion Date

May 31, 2016

Study Completion Date

May 31, 2016

Conditions

MPS 7Sly SyndromeMucopolysaccharidosisMPS VII

Interventions

DRUG

UX003

UX003 is a sterile concentrate formulation of rhGUS for intravenous infusion

OTHER

Placebo

Placebo consisting of the UX003 formulation buffer (without rhGUS)

Trial Locations (4)

33155

Miami Children's Hospital, Miami

55455

University of Minnesota, Minneapolis

92868

Children's Hospital of Orange County, Orange

94609

Children's Hospital Oakland, Oakland

All Listed Sponsors

lead

Ultragenyx Pharmaceutical Inc

INDUSTRY

NCT02230566 - A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) | Biotech Hunter | Biotech Hunter