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: 07 Feb 2018

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

    • 07 Feb 2018 Interim results were presented in a BioMarin Pharmaceutical media release.
    • 07 Feb 2018 According to a BioMarin Pharmaceutical media release, interim data from this trial was presented at WORLDSymposium 2018.
    • 05 Feb 2018 According to a BioMarin Pharmaceutical media release, data will be presented at the 14th Annual WORLDSymposiumTM 2018.
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