Active not recruitingPHASE1, PHASE2INTERVENTIONAL

Gene Therapy Study for Children With CLN5 Batten Disease

Researchers are conducting a study to test a new gene therapy, NGN-101, for children between 3 and 9 years old who have CLN5 Batten disease. This treatment aims to replace a faulty gene that causes the disease. The study will specifically look at how safe the treatment is and if it can improve children's movement, speech, vision, and thinking abilities. It's an early-stage study, meaning it's one of the first times this treatment is being given to people. Participants will receive the treatment once and then be monitored closely for 5 years to see its long-term effects. The hope is to find a way to help manage this serious condition.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Neurogene Inc.

Enrolment target

Start

31 Jan 2022

Estimated completion

01 Nov 2028

Neuronal Ceroid Lipofuscinosis CLN5

What is this study about?

Batten disease, especially the CLN5 type, is a serious condition that affects children and can lead to problems with movement, speech, vision, and thinking. This study is exploring a new type of treatment called 'gene therapy' for children with CLN5 Batten disease. Gene therapy works by delivering a healthy copy of the CLN5 gene into the body to replace the faulty one that causes the disease. The specific treatment being tested is called NGN-101.

This study is being done in two early phases (Phase 1 and Phase 2), which means it's one of the first times this gene therapy is being given to people. The main goals are to find out if NGN-101 is safe for children and to see if it can help improve their symptoms or slow down the disease's progression. Researchers will be looking at how the treatment affects different aspects of the children's health, including their ability to move, talk, see, and think.

The children taking part will receive the NGN-101 treatment once. This treatment involves delivering the gene therapy directly into the fluid around the brain and also into the eyes. After the treatment, the children will be carefully monitored for a full 5 years. This long follow-up period is important to understand the treatment's safety and how well it works over time.

Key takeaways

It's a gene therapy study for children aged 3-9 with CLN5 Batten disease.
The treatment (NGN-101) is given once via injections to the brain fluid and eyes.
The main goals are to check for safety and whether it helps with symptoms.
Children will be monitored for 5 years after receiving the treatment.
This is an early-stage study, so the benefits and risks are still being understood.

Who may be eligible?

This study is specifically looking for children between 3 and 9 years old who have been diagnosed with CLN5 Batten disease by a doctor and through genetic testing. They should also be experiencing some problems with their movement, speech, or eyesight.

To be considered for the study, children must be able to manage certain medical tests, which might involve a stay in the hospital, and potentially require medicine to help them relax or sleep during the procedures. They should also be able to walk, perhaps with some help like a walker or holding someone's hand. Their parent or legal guardian will need to give written permission, and the child will also need to agree to take part if they are able.

Children generally won't be able to join if they have other serious brain conditions, infections, or other health problems that would make the study procedures unsafe. Also, if they have certain types of seizures that are frequent or severe, or have received other experimental treatments recently, they might not be suitable for this study.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is your child aged between 3 and 9 years old?
Does your child have a confirmed diagnosis of CLN5 Batten disease?
Does your child have problems with movement, speech, or vision?
Can your child manage medical tests that might require sedation or general anaesthetic?
Are you able to live within an hour's drive of the study site for at least 6 months after treatment?
Does your child have any other severe or recent illnesses?

Answer every question to see your result.

What does participation involve?

If your child qualifies and you decide to take part, they will receive the NGN-101 gene therapy once. This treatment involves injections into the fluid around the brain and into their eyes, both on the same day. These procedures will require a stay in the hospital and they might need medicine to help them relax or sleep comfortably.

After getting the treatment, your child will have regular check-ups and tests for 5 years. These checks will include various assessments to monitor their safety and to see if the treatment is having any effect on their movement, language, vision, and thinking skills. You will need to live within an hour's drive of the study site for at least the first 6 months after treatment to allow for regular visits. The total duration of active follow-up for safety and how well the treatment works will be 5 years.

Potential risks and benefits

As with any new treatment, there are potential benefits and risks. The potential benefits include the possibility that NGN-101 could improve your child's symptoms or slow down the progression of CLN5 Batten disease, as existing treatments just manage symptoms. However, because this is an early-stage study, we don't yet know how effective it will be. Potential risks include side effects from the gene therapy itself or from the procedures needed to give the treatment, such as injections and sedation. You will be given full details about known and potential risks. It's important to remember that participation is voluntary, and you have the right to withdraw your child from the study at any time, for any reason, without it affecting their future medical care.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of Rochester
Verified postcode
Rochester, United States
Great Ormond Street Hospital for Children
Verified postcode
London, United Kingdom

Common questions

What is CLN5 Batten disease?

It's a serious genetic condition that affects the brain and nervous system, leading to problems with movement, speech, vision, and thinking abilities in children.

What is NGN-101?

NGN-101 is a new gene therapy treatment being tested. It aims to deliver a healthy copy of a gene to help correct the underlying cause of CLN5 Batten disease.

How is the treatment given?

The treatment is given once through injections into the fluid surrounding the brain and directly into the eyes, both on the same day.

How long will the study last for my child?

Your child will receive the single treatment and then be monitored closely for 5 years to check on their safety and how well the treatment is working.

Can my child stop participating if we change our minds?

Yes, you have the right to withdraw your child from the study at any time without it affecting their usual medical care.