Trial Profile

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

Status: Active, no longer recruiting

Phase of Trial: Phase I/II

Latest Information Update: 14 Aug 2024

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At a glance

Drugs NGN-101 (Primary) ; NGN-101 (Primary)
Indications Neuronal ceroid lipofuscinosis
Focus Adverse reactions; First in man
Acronyms CLN5-200
Sponsors Neurogene Inc

Most Recent Events

12 Aug 2024 Status changed from recruiting to active, no longer recruiting.
09 Aug 2024 According to a Neurogene media release, plans to provide interim clinical data and a regulatory update in the first quarter of 2025.
05 Jan 2024 According to a Neurogene media release, Neurogene has completed enrollment of Cohorts 1 and 2 in this study and is currently enrolling a final higher dose cohort.

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