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: 02 Aug 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

    • 02 Aug 2017 According to a BioMarin media release, the study has now moved to the expansion phase with the highest dose and measuring changes in neurocognitive function in children with this rapid andprogressive neurodegenerative disease.
    • 02 Aug 2017 Results published in the BioMarin media release.
    • 10 Jun 2017 Biomarkers information updated
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