Gene Therapy for Batten Disease

(CLN5-200 Trial)

Recruiting at 1 trial location

Overseen By Contact Center

Age: < 18

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Neurogene Inc.

Must not be taking: Prohibited medications

Disqualifiers: Other neurologic disease, Seizure mutation, Active infections, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 1 day

6 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial mentions the use of prohibited medications, but it does not specify which ones. It's best to discuss your current medications with the study team to see if any need to be stopped.

What data supports the effectiveness of the treatment NGN-101 for Batten Disease?

Research on similar gene therapies for Batten disease shows promising results. For example, a gene therapy for CLN7 Batten disease improved symptoms and survival in mice, and another therapy for CLN6 Batten disease increased lifespan and improved motor skills in mice. These findings suggest that gene therapy could be effective for treating Batten disease. ^{1 2 3 4 5}

Is gene therapy for Batten disease safe in humans?

Gene therapy for Batten disease has shown an acceptable safety profile in animal studies, including mice and rats, and was well tolerated in non-human primates. In a small human trial for a related form of Batten disease, expected adverse events occurred but did not cause long-term disability. ^{1 2 3 4 5}

How is the treatment NGN-101 for Batten Disease different from other treatments?

NGN-101 is a gene therapy specifically targeting the CLN7 form of Batten disease, which is an ultra-rare condition with no existing treatments that can stop its progression. This therapy uses an adeno-associated virus (AAV) to deliver a healthy copy of the CLN7 gene, showing promising results in animal models by improving symptoms and survival, which is a novel approach compared to traditional treatments. ^{1 2 4 5 6}

What is the purpose of this trial?

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.

Research Team

Effie Albanis, MD

Principal Investigator

Neurogene Inc.

Eligibility Criteria

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.

Inclusion Criteria

I can walk, even if I need some help or use a device.

I can undergo tests like blood tests, MRI, and others that may need sedation.

My condition is due to a CLN5 gene mutation.

Exclusion Criteria

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.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Single administration of gene therapy via intracerebroventricular and intravitreal injection

1 day

Follow-up

Participants are monitored for safety and efficacy, including assessments of motor, language, visual, and cognitive function

5 years

Multiple visits

Treatment Details

Interventions

• NGN-101

Trial Overview 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.

Participant Groups

Group I: Cohort 3 Experimental Treatment 1 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 2 Experimental Treatment 1 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 1 Experimental Treatment 1 Intervention

The study treatment is a recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt).

Who Is Running the Clinical Trial?

Neurogene Inc.

Lead Sponsor

Trials

6

Recruited

150+

References

1. United States pubmed.ncbi.nlm.nih.gov

CLN7 gene therapy: hope for an ultra-rare condition. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Therapeutic landscape for Batten disease: current treatments and future prospects. [2023]

3. England pubmed.ncbi.nlm.nih.gov

Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Isolation and chromosomal mapping of a mouse homolog of the Batten disease gene CLN3. [2007]