A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

Trial Profile

A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 24 Dec 2016

At a glance

  • Drugs Lesinidase alfa (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Adverse reactions; First in man
  • Sponsors Alexion Pharmaceuticals; Synageva BioPharma
  • Most Recent Events

    • 07 Jun 2017 Biomarkers information updated
    • 27 Oct 2016 According to an Alexion media release, planned dose escalation, with all patients randomized to either a 5 mg/kg or 10 mg/kg dose has been completed
    • 14 Jul 2016 According to an Alexion media release, data from this trial were presented at the 14th International Symposium on MPS and Related Diseases
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