A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

Trial Profile

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 11 Dec 2017

At a glance

  • Drugs BMN 250 (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Acronyms MPS IIIB
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 06 Sep 2017 Results published in a BioMarin Pharmaceutical Media Release
    • 06 Sep 2017 Interim data from the dose escalation arm of this study presented at the 13th International Congress of Inborn Errors of Metabolism (ICIEM) 2017, according to a BioMarin Pharmaceutical media release.
    • 05 Sep 2017 According to a BioMarin Pharmaceutical media release, data will be presented at the 13th International Congress of Inborn Errors of Metabolism (ICIEM) 2017.
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