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: 31 Jul 2017

At a glance

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

    • 01 Jun 2017 According to a BioMarin Pharmaceutical media release, the European Commission (EC) has granted marketing authorization for Brineura (cerliponase alfa), the first treatment approved in the European Union for the treatment of (CLN2). The MAA was based on this and an open-label extension study.
    • 27 Apr 2017 According to an FDA media release, the US FDA approved Brineura (cerliponase alfa) as a treatment for a specific form of Batten disease. Brineura is the first FDA-approved treatment to slow loss of walking ability (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.
    • 06 Dec 2016 Results of this study assessing safety, tolerability and efficacy of every other week intracerebroventricular infusions of cerliponase alfa in children with CLN2 aged 3-16 years, presented at the 70th Annual Meeting of the American Epilepsy Society.
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