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A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

Trial Profile

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

Completed
Phase of Trial: Phase I/II

Latest Information Update: 14 Jun 2018

At a glance

  • Drugs Cerliponase alfa (Primary)
  • Indications Neuronal ceroid lipofuscinosis
  • Focus First in man; Therapeutic Use
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 31 Aug 2018 Biomarkers information updated
    • 24 Apr 2018 This study was performed in accordance with the Declaration of Helsinki; the study protocol was approved by relevant ethics boards.
    • 24 Apr 2018 Updated results published in the New England Journal of Medicine (NEJM) in the May 2018 issue, according to a BioMarin Pharmaceutical media release
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