Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

September 11, 2023 updated by: David L Rogers, MD

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

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

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

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

Enrollment (Estimated)

5

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 6 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Genotypic confirmation of classical CLN2 Batten's disease from a CLIA certified lab.
  • Enzyme level deficiency of tripeptidyl-peptidase
  • Minimum age requirement: 24 months of age at enrollment
  • Maximum age requirement: 72 months of age at enrollment
  • Currently receiving intraventricular cerliponase alfa
  • Willing to participate in the proposed study visits over the 2-year period
  • Minimum central retinal thickness (CRT) of 140μm based upon OCT assessment
  • Clear ocular media
  • No ocular pathology present to account for vision loss other than optic atrophy and pigmentary retinopathy that is felt to be due to the CLN2 disease process

Exclusion Criteria:

  • Any opacities in the clear ocular media including vitreous debris.
  • History of ocular trauma or prior ocular surgery.
  • Episode of generalized motor status epilepticus within four weeks before the First Dose visit
  • Severe infection (e.g., upper respiratory tract infection, pneumonia, pyelonephritis, or meningitis) within four weeks before the First Dose visit (enrollment may be postponed)
  • Those with a history of bleeding disorders.
  • History of or current chemotherapy, radiotherapy or other immunosuppression therapy within the past 30 days (corticosteroid treatment may be permitted at the discretion of the PI)
  • Has a medical condition, or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with the protocol required testing or procedures or compromise the subject's wellbeing, safety, or clinical interpretability

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Intervention
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.
Other Names:
  • Brineura

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Monitoring for the development of unacceptable toxicity.
Time Frame: 2 years
Based on the development of unacceptable toxicity, defined as the occurrence of any Grade 3 or higher, unanticipated, treatment related toxicity.
2 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Efficacy of intravitreal cerliponase alfa to stabilize retinal architecture.
Time Frame: 2 years
Efficacy will be determined by measuring central retinal thickness via OCT imaging prior to each injection.
2 years
Efficacy of intravitreal cerliponase alfa to stabilize fundoscopic features.
Time Frame: 2 years
Efficacy will be determined by measuring the Weill Cornell LINCL Ophthalmic Severity Score prior to each injection.
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

David L Rogers, MD

Investigators

  • Principal Investigator: David Rogers, MD, Nationwide Children's Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 1, 2021

Primary Completion (Estimated)

November 1, 2025

Study Completion (Estimated)

November 1, 2027

Study Registration Dates

First Submitted

November 29, 2021

First Submitted That Met QC Criteria

November 29, 2021

First Posted (Actual)

December 10, 2021

Study Record Updates

Last Update Posted (Actual)

September 13, 2023

Last Update Submitted That Met QC Criteria

September 11, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • STUDY00001444

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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