An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Trial Profile

An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Completed
Phase of Trial: Phase I/II

Latest Information Update: 20 Mar 2017

At a glance

  • Drugs Vestronidase alfa (Primary)
  • Indications Mucopolysaccharidosis VII
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Sponsors Ultragenyx Pharmaceutical
  • Most Recent Events

    • 06 Mar 2017 Status changed from active, no longer recruiting to completed.
    • 18 May 2016 The trial was completed in Spain.
    • 10 Feb 2015 Results presented at 11th WORLD Symposium, according to Ultragenyx media release.
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