Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

David L Rogers, MD

Status and phase

Active, not recruiting

Phase 2

Phase 1

Conditions

Neuronal Ceroid Lipofuscinosis Type 2

Treatments

Drug: Cerliponase Alfa

Study type

Interventional

Funder types

Other

Identifiers

NCT05152914

STUDY00001444

Details and patient eligibility

About

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

Full 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.

Enrollment

5 estimated patients

Sex

All

Ages

24 to 72 months old

Volunteers

No Healthy Volunteers

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