NCT01907087 - 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 (CLN2) Disease | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

September 30, 2013

Primary Completion Date

November 30, 2015

Study Completion Date

November 30, 2015

Conditions

Jansky-Bielschowsky DiseaseBatten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2CLN2 Disease

Interventions

BIOLOGICAL

BMN 190

30-300 mg ICV infusion administered every other week for at least 48 weeks.

Trial Locations (5)

University Hamburg-Eppendorf, Hamburg

Nationwide Children's Hospital, Columbus

Bambino Gesù Children's Hospital, Rome

Guy's & St. Thomas NHS Foundation Trust, London

Great Ormond Street Hospital for NHS Foundation Trust, London