A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/Week and 2.0 mg/kg/Every Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Trial Profile

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/Week and 2.0 mg/kg/Every Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Completed
Phase of Trial: Phase III

Latest Information Update: 30 Sep 2017

At a glance

  • Drugs Elosulfase alfa (Primary)
  • Indications Mucopolysaccharidosis IV
  • Focus Registrational; Therapeutic Use
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 01 Nov 2014 Primary endpoint (Change From Baseline in Endurance as Measured by the 6-minute Walk Test) has been met as results published in the Journal of Inherited Metabolic Disease
    • 26 Feb 2014 New trial record
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