Status:
ACTIVE_NOT_RECRUITING
Gene Therapy Study for Children With CLN5 Batten Disease
Lead Sponsor:
Neurogene Inc.
Conditions:
Neuronal Ceroid Lipofuscinosis CLN5
Eligibility:
All Genders
3-9 years
Phase:
PHASE1
PHASE2
Brief 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) Su...
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 ge...
Eligibility Criteria
Inclusion
- Inclusion Criteria
- 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)
- Exclusion Criteria
- 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
Exclusion
Key Trial Info
Start Date :
January 31 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 1 2028
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT05228145
Start Date
January 31 2022
End Date
November 1 2028
Last Update
August 12 2024
Active Locations (2)
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1
University of Rochester
Rochester, New York, United States, 14642
2
Great Ormond Street Hospital for Children
London, United Kingdom, WC1N 3JH