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Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

NCT05152914 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2026-04-30

No results posted yet for this study

Summary

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

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.

Sponsors & Collaborators

  • David L Rogers, MD

    lead OTHER

Principal Investigators

  • David Rogers, MD · Nationwide Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
24 Months
Max Age
72 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-11-01
Primary Completion
2026-03-04
Completion
2027-03-06
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05152914 on ClinicalTrials.gov