An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

23

Participants

Timeline

Start Date

February 28, 2015

Primary Completion Date

December 10, 2020

Study Completion Date

December 10, 2020

Conditions
Jansky-Bielschowsky DiseaseBatten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2CLN2 DiseaseCLN2 Disorder
Interventions
BIOLOGICAL

BMN 190

300 mg Intracerebroventricular (ICV) infusion administered every other week for up to 240 weeks

DEVICE

Intracerebroventricular (ICV) access device

Surgical implantation of an MRI compatible ICV access device in the lateral ventricle of the right hemisphere is required for administration of study drug.

Trial Locations (4)

20246

Universitaetsklinikum Hamburg-Eppendorf, Hamburg

43205

Nationwide Children's Hospital, Columbus

00165

Children's Hospital Bambino Gesù,IRCCS, Rome

WC1N 3JH

Great Ormond Street Childrens Hospital, London

Sponsors
All Listed Sponsors
lead

BioMarin Pharmaceutical

INDUSTRY