NCT05228145View on ClinicalTrials.gov

Gene Therapy Study for Children With CLN5 Batten Disease

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

January 31, 2022

Primary Completion Date

November 30, 2028

Study Completion Date

November 30, 2028

Conditions
Neuronal Ceroid Lipofuscinosis CLN5
Interventions
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.

Trial Locations (2)

14642

University of Rochester, Rochester

WC1N 3JH

Great Ormond Street Hospital for Children, London

Sponsors

Lead Sponsor

Neurogene Inc.
All Listed Sponsors
lead

Neurogene Inc.

INDUSTRY

NCT05228145 - Gene Therapy Study for Children With CLN5 Batten Disease | Biotech Hunter | Biotech Hunter