A Phase 1/2 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis (NCL) Subtype 5 (CLN5) Disease
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.
• Age from 3 to 9 years (Child)
• Molecular genetic diagnosis of the CLN5 gene
• Confirmed clinical diagnosis of CLN5 disease
• Impaired motor and/or language function and/or impaired visual acuity
• Written informed consent from parent or legal guardian and assent from study participant, if appropriate
• Able to comply with protocol required assessments (laboratory sample collection, lumbar puncture (LP), nerve conduction studies (NCS), magnetic resonance imaging (MRI), etc.), which may require sedation or general anesthesia
• Able to walk with or without assistance (assistance may include a walker, braces, or with one hand held)
• Agree to reside within a 1-hour drive of the study site for at least 6 months following treatment (or a safely drivable distance for the study participant and caregivers according to investigator's discretion)