A Phase 1/2 interventional study of NGN-101 in Neuronal Ceroid Lipofuscinosis CLN5, sponsored by Neurogene Inc.. Active, not recruiting at 2 sites in 2 countries. Open to participants aged 3 Years to 9 Years. Per ClinicalTrials.gov, last updated 2024-08-12.
Sponsored by Neurogene Inc. · Phase 1/2, Interventional, and Treatment
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.
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 gene encoding human ceroid-lipofuscinosis neuronal protein 5 (CLN5) in subjects with CLN5 Batten disease. The study treatment will be delivered via intracerebroventricular (ICV) and intravitreal (IVT) injection on the same day. Each participant will be followed for safety and efficacy for 5 years after treatment. Efficacy assessments in this study will evaluate motor, language, visual and cognitive function.
49 studies on the registry are indexed under Neuronal Ceroid-Lipofuscinoses; 13 are open to participants now.
This study's enrollment of 6 is below the median of 13 across 22 interventional studies indexed under Neuronal Ceroid-Lipofuscinoses.
Browse Neuronal Ceroid-Lipofuscinoses studies →Neurogene Inc. is the lead sponsor of 6 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The study treatment is a recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt).
Genetic: NGN-101
The study treatment is a higher dose of recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt).
Genetic: NGN-101
The study treatment is a higher dose of recombinant serotype 9 adeno-associated virus encoding a codon- optimized human CLN5 transgene (hCLN5opt).
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.
Incidence of Treatment Emergent Adverse Events (TEAEs)
Incidence, type, severity, and frequency of TEAEs
Time frame: 5 years (multiple visits)
Incidence of Serious Adverse Events (SAEs)
Incidence, type, severity, and frequency of SAEs
Time frame: 5 years (multiple visits)
Incidence of clinical laboratory abnormalities
Incidence, type, severity, and frequency of clinical laboratory abnormalities
Time frame: 5 years (multiple visits)
Incidence of new nerve conduction study (NCS) abnormalities
Incidence, type, severity, and frequency of new nerve conduction study (NCS) abnormalities
Time frame: 5 years (multiple visits)
Incidence of new physical and neurologic exam abnormalities
Incidence, type, severity, and frequency of new physical and neurologic exam abnormalities
Time frame: 5 years (multiple visits)
Change in Hamburg Scale, Motor and Language domain scores
Change from baseline in Hamburg Scale, Motor and Language domain scores (each domain is rated from 0 to 3, with 3 reflecting normal function for age and 0 reflecting complete loss of function)
Time frame: 5 years (multiple visits)
Change in Spectral Domain-Optical Coherence Tomography (SD-OCT)
Change from baseline in SD-OCT parameters including Ellipsoid Zone (EZ) defect area measurements, macular volume and thickness, retinal nerve fiber layer thickness, and ganglion cell layer thickness
Time frame: 5 years (multiple visits)
Change in Unified Batten Diseases Rating Scale (UBDRS)
Change from baseline in total score and individual domains of the Unified Batten Diseases Rating Scale (UBDRS; total score 0 to 277, with higher scores indicating worse function)
Time frame: 5 years (multiple visits)
Change in Caregiver global impression of change
Caregiver global impression of change throughout the study
Time frame: 5 years (multiple visits)
Change in visual acuity measurements
Change from baseline in visual acuity measured using Teller acuity cards, Lea symbol chart, Landolt C chart, or low contrast visual acuity (measure to be used will depend on subject's level of cognitive and visual function)
Time frame: 5 years (multiple visits)
Change in color vision
Change from baseline in color vision measured using Ishihara color blindness testing
Time frame: 5 years (multiple visits)
Plan to share: No
This study is active, not recruiting, as verified in Aug 2024. You cannot join it, but the record below documents what was studied.
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Neuronal Ceroid-Lipofuscinoses →
Neurogene Inc.