This is a phase I/II randomized, masked, clinical trial to determine the safety and efficacy of intravitreal administration of cerliponase alfa.
This is a Phase I/II study for 5 subjects receiving an intravitreal injection of cerliponase alfa under sedation into the proclaimed study eye(s) in a 4-week interval over 24 months. This study will be monitored by a Data Safety Monitoring Committee (DSMB). Each subject will participate in the ongoing study for an active period of 2 years. Subjects will then transfer to a bi-annual monitoring program where data will be collected from bi-annual standard of care visits for an additional 3 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
5
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.
Nationwide Children's Hospital
Columbus, Ohio, United States
Monitoring for the development of unacceptable toxicity.
Based on the development of unacceptable toxicity, defined as the occurrence of any Grade 3 or higher, unanticipated, treatment related toxicity.
Time frame: 2 years
Efficacy of intravitreal cerliponase alfa to stabilize fundoscopic features.
Efficacy will be determined by measuring the Weill Cornell LINCL Ophthalmic Severity Score prior to each injection.
Time frame: 2 years
Efficacy of intravitreal cerliponase alfa to stabilize retinal architecture.
Efficacy will be determined by measuring central retinal thickness via OCT imaging prior to each injection.
Time frame: 2 years
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