Your session is about to expire
← Back to Search
Gene Therapy
Gene Therapy for Batten Disease (CLN5-200 Trial)
Phase 1 & 2
Waitlist Available
Research Sponsored by Neurogene Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Molecular genetic diagnosis of the CLN5 gene
Impaired motor and/or language function and/or impaired visual acuity
Must not have
Known pathogenic or clinically suspected variant in a seizure associated genetic mutation besides CLN5
Any anticipated need for major surgery in the next 24 months
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years (multiple visits)
Awards & highlights
No Placebo-Only Group
Summary
This trial tests a gene therapy that uses a harmless virus to deliver a healthy gene to children with a rare genetic disorder affecting their nervous system. The virus helps replace the faulty gene causing the disease.
Who is the study for?
This trial is for children aged 3 to 9 with a confirmed genetic diagnosis of CLN5 Batten Disease who can walk (with or without help) and comply with study procedures like MRI scans. They must not have been in another drug study recently, have certain infections or allergies, need major surgery soon, or be on ventilatory support.
What is being tested?
The trial tests NGN-101 gene therapy in a single dose escalation format. It's an open-label study where all participants receive the treatment. The aim is to see how different doses are tolerated and how effective they are against CLN5 Batten Disease symptoms.
What are the potential side effects?
Potential side effects may include reactions related to the immune system due to gene therapy, discomfort from procedures required by the trial such as lumbar puncture or MRI under sedation/anesthesia, and general risks associated with gene therapies.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My condition is due to a CLN5 gene mutation.
Select...
I have difficulties with movement, speaking, or seeing clearly.
Select...
I am between 3 and 9 years old.
Select...
I have been diagnosed with CLN5 disease.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I have a genetic mutation linked to seizures, not including CLN5.
Select...
I do not expect to need major surgery in the next 2 years.
Select...
I have not received any vaccines in the last 45 days.
Select...
I do not have any health issues that prevent me from undergoing procedures like spinal taps or using anesthesia.
Select...
I haven't had chemotherapy, radiotherapy, or immunosuppressive therapy in the last 3 months.
Select...
I am allergic to the drugs used in the immunosuppression treatment.
Select...
I do not have any health conditions that prevent eye injections.
Select...
I need a machine to help me breathe during the day or night.
Select...
I am not taking any medications that are not allowed in the study.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 5 years (multiple visits)
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years (multiple visits)
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Incidence of Serious Adverse Events (SAEs)
Incidence of Treatment Emergent Adverse Events (TEAEs)
Incidence of clinical laboratory abnormalities
+2 moreSecondary study objectives
Change in Caregiver global impression of change
Change in Hamburg Scale, Motor and Language domain scores
Change in Spectral Domain-Optical Coherence Tomography (SD-OCT)
+3 moreAwards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
3Treatment groups
Experimental Treatment
Group I: Cohort 3Experimental Treatment1 Intervention
The study treatment is a higher dose of recombinant serotype 9 adeno-associated virus encoding a codon- optimized human CLN5 transgene (hCLN5opt).
Group II: Cohort 2Experimental Treatment1 Intervention
The study treatment is a higher dose of recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt).
Group III: Cohort 1Experimental Treatment1 Intervention
The study treatment is a recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt).
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Gene therapy for Neuronal Ceroid Lipofuscinosis (NCL) involves the introduction, removal, or alteration of genetic material to correct the underlying genetic defects causing the disease. This approach aims to restore the function of deficient enzymes or proteins that are crucial for cellular health.
For instance, in CLN5 Batten disease, gene therapy introduces a functional copy of the defective gene to produce the necessary enzyme, thereby reducing the accumulation of toxic substances in cells. This is particularly important for NCL patients as it addresses the root cause of the disease, potentially halting or reversing the progression of neurodegeneration and improving quality of life.
Experimental Therapeutic Approaches for the Treatment of Retinal Pathology in Neuronal Ceroid Lipofuscinoses.Gene therapy: prospects for glycolipid storage diseases.
Experimental Therapeutic Approaches for the Treatment of Retinal Pathology in Neuronal Ceroid Lipofuscinoses.Gene therapy: prospects for glycolipid storage diseases.
Find a Location
Who is running the clinical trial?
Neurogene Inc.Lead Sponsor
5 Previous Clinical Trials
141 Total Patients Enrolled
Effie Albanis, MDStudy DirectorNeurogene Inc.
Xiomara Q. Rosales, MDStudy DirectorNeurogene Inc.
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I can walk, even if I need some help or use a device.I've had seizures lasting over 5 minutes or multiple seizures within 5 minutes without fully recovering in the last 12 weeks.You have a high level of antibodies against AAV9 in your blood.I have a genetic mutation linked to seizures, not including CLN5.I do not expect to need major surgery in the next 2 years.I can undergo tests like blood tests, MRI, and others that may need sedation.My condition is due to a CLN5 gene mutation.I have not received any vaccines in the last 45 days.I have difficulties with movement, speaking, or seeing clearly.I do not have any health issues that prevent me from undergoing procedures like spinal taps or using anesthesia.I am between 3 and 9 years old.I have a neurological condition that could have caused cognitive issues before joining the study.I haven't had chemotherapy, radiotherapy, or immunosuppressive therapy in the last 3 months.I am allergic to the drugs used in the immunosuppression treatment.I do not have any health conditions that prevent eye injections.I need a machine to help me breathe during the day or night.I have been diagnosed with CLN5 disease.I have not had any severe infections in the last 30 days.I am not taking any medications that are not allowed in the study.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 2
- Group 2: Cohort 1
- Group 3: Cohort 3
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.