Gene Therapy Study for Children With CLN5 Batten Disease

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Neuronal Ceroid Lipofuscinosis CLN5

In brief

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.

Key facts

Study ID

NCT05228145

Run by

Neurogene Inc.

People needed

6

Starts

2022-01-31

Expected to finish

2028-11-01

Last updated by the study team

2024-08-12

Who can join

Age: 3 and older, up to 9. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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)

You may not qualify if…

• Has another neurologic disease or illness that may have caused cognitive decline before study entry
• Known pathogenic or clinically suspected variant in a seizure associated genetic mutation besides CLN5
• Any active infections or severe infections within the 30 days prior to study treatment administration
• Presence of a concomitant medical condition that precludes intracerebroventricular (ICV) injection, lumbar puncture (LP), or use of anesthetics needed for study-related procedures
• Presence of any concomitant medical conditions that preclude intravitreal (IVT) administration
• Has status epilepticus that lasts longer than 5 minutes or having more than 1 seizure within a 5-minute period, without returning to a normal level of consciousness between episodes within 12 weeks before study treatment
• Total anti-AAV9 antibody titer greater than 1:400
• Any anticipated need for major surgery in the next 24 months
• Participation in an Investigational New Drug, Investigational Device Exemption, or equivalent clinical study in the past 6 months
• Any prior participation in a study in which a gene therapy vector or stem cell transplantation was administered
• Participation in other investigational studies and non-interventional studies that have similar study assessments as this protocol while the study participant is enrolled in this study with the exception of sister studies sponsored by Neurogene
• History of or current chemotherapy, radiotherapy, or other immunosuppressive therapy within the past 3 months
• Use of prohibited medications
• Immunizations of any kind in the 45 days prior to study treatment
• Requiring daytime or nighttime ventilatory support at the time of Screening
• Any item which would exclude the study participant from being able to undergo brain magnetic resonance imaging (MRI) according to local institutional policy
• Known allergies or hypersensitivities to the required immunosuppression regime

Where it is running

• University of Rochester — Rochester, New York, United States
• Great Ormond Street Hospital for Children — London, United Kingdom

Full record on ClinicalTrials.gov

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