NCT05228145

Gene Therapy Study for Children With CLN5 Batten Disease

A Phase 1/2 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis (NCL) Subtype 5 (CLN5) Disease

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Neurogene Inc. · Industry
• Sex: All
• Age: 3 Years – 9 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective, non-randomized, open-label, dose escalation study of a single administration of gene therapy in children who are 3 to 9 years old with Neuronal Ceroid Lipofuscinosis (Batten) Subtype 5 (CLN5) disease.

Detailed description

The study is a first in human (FIH) open-label, dose escalation study designed to assess the safety and efficacy of administration of an adeno-associated viral vector serotype 9 (AAV9) carrying the gene encoding human ceroid-lipofuscinosis neuronal protein 5 (CLN5) in subjects with CLN5 Batten disease. The study treatment will be delivered via intracerebroventricular (ICV) and intravitreal (IVT) injection on the same day. Each participant will be followed for safety and efficacy for 5 years after treatment. Efficacy assessments in this study will evaluate motor, language, visual and cognitive function.

Conditions

• Neuronal Ceroid Lipofuscinosis CLN5

Interventions

Type	Name	Description
GENETIC	NGN-101	Participants with confirmed mutations in the CLN5 gene who meet all the inclusion and none of the exclusion criteria will be treated with a single intracerebroventricular (ICV) dose and a single intravitreal (IVT) dose of the study treatment.

Timeline

• Start date: 2022-01-31
• Primary completion: 2028-11-01
• Completion: 2028-11-01
• First posted: 2022-02-08
• Last updated: 2024-08-12

Locations

2 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study