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ACTIVE NOT RECRUITING
NCT05152914
PHASE1/PHASE2

Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

Sponsor: David L Rogers, MD

View on ClinicalTrials.gov

Summary

This is a phase I/II randomized, masked, clinical trial to determine the safety and efficacy of intravitreal administration of cerliponase alfa.

Official title: Intravitreal Enzyme Replacement Therapy to Prevent Retinal Disease Progression in Children With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)

Key Details

Gender

All

Age Range

24 Months - 72 Months

Study Type

INTERVENTIONAL

Enrollment

5

Start Date

2021-11-01

Completion Date

2027-11-01

Last Updated

2025-06-11

Healthy Volunteers

No

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.

Locations (1)

Nationwide Children's Hospital

Columbus, Ohio, United States