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Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2
Sponsor: David L Rogers, MD
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
Conditions
Interventions
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