Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

5

Participants

Timeline

Start Date

November 1, 2021

Primary Completion Date

November 1, 2025

Study Completion Date

November 1, 2027

Conditions
Neuronal Ceroid Lipofuscinosis Type 2
Interventions
DRUG

Cerliponase Alfa

Brineura is a hydrolytic lysosomal N-terminal tripeptidyl peptidase indicated to slow the loss of 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.

Trial Locations (1)

43205

Nationwide Children's Hospital, Columbus

All Listed Sponsors
lead

David L Rogers, MD

OTHER