A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

PHASE2CompletedINTERVENTIONAL
Enrollment

14

Participants

Timeline

Start Date

January 22, 2016

Primary Completion Date

April 20, 2022

Study Completion Date

April 20, 2022

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

BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)

DEVICE

Intracerebroventricular 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