AuthorisedPhase I and Phase II (Integrated)- First administration to humansInterventional

A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy with TTX-381 for the Ocular Manifestations Associated with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

This research is looking at a new treatment called TTX-381 for children who have a rare genetic condition called Neuronal Ceroid Lipofuscinosis Type 2 (CLN2 disease). This condition can cause serious eye problems, leading to vision loss. This is the very first time this gene therapy is being given to humans, so it's a very early stage of testing. The main goals are to check if the treatment is safe and if people can tolerate it well over about a year. Doctors will also be closely watching to see if it helps improve or slow down the eye issues associated with CLN2 disease. If successful, this could offer a new way to help children living with this challenging condition.

At a glance

Status

Authorised

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Tern Therapeutics LLC

Enrolment target

Start

03 Jul 2025

Ocular manifestations of Neuronal Ceroid Lipofuscinosis (CLN) Type 2

What is this study about?

This study is about a new and hopeful treatment for a serious condition called Neuronal Ceroid Lipofuscinosis Type 2, often shortened to CLN2 disease. This is a rare genetic disorder that affects children. Over time, it causes problems with the brain, movement, and sadly, their eyesight. The eye problems can get worse, leading to severe vision loss.

The new treatment being tested is a 'gene therapy' called TTX-381. Gene therapy tries to fix the root cause of certain diseases by introducing healthy genetic material into the body. Think of it like giving the body missing instructions it needs to work properly. In this case, TTX-381 is designed to specifically target the eye and potentially help improve or protect the vision of children with CLN2 disease.

This is a very important step because it's the first time this particular gene therapy is being given to people. This early stage of testing is mainly focused on checking if the treatment is safe and if people tolerate it without too many side effects. Doctors will also be looking closely to see if there are any signs that it is helping with the eye problems caused by CLN2 disease, and how long any effects might last. The hope is that this research could eventually lead to a new treatment option for children who desperately need it.

Key takeaways

This study is testing a new gene therapy (TTX-381) for eye problems in CLN2 disease.
It's the first time this treatment is being given to people.
The main goal is to check for safety and how well people tolerate the treatment.
Researchers will also look for signs that vision might be improving or stabilising.
The study lasts about one year, with regular clinic visits.
This research hopes to find new ways to help children with CLN2.

Who may be eligible?

This study is open to both boys and girls who have been diagnosed with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2 disease). There are no specific age limits mentioned, meaning that children of various ages with CLN2 disease could potentially be considered.

To be clear, this study is specifically for individuals with CLN2 disease who are experiencing eye-related issues as a result of their condition. The research team will review each potential participant carefully to make sure the study is a good fit for them.

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.

I (or my child) have been diagnosed with CLN2 disease.
I (or my child) am experiencing eye problems linked to CLN2 disease.
I understand this is a very early stage (first-in-human) study.
I am willing and able to attend regular clinic appointments for about a year.

Answer every question to see your result.

What does participation involve?

Taking part in this study means you or your child would receive the new gene therapy. You would have regular visits to the clinic for about one year (360 days). During these visits, doctors and nurses would perform various checks. This includes detailed eye exams, blood tests, and possibly collecting samples of tears or spinal fluid (called aqueous humor and CSF) to see how the treatment is working in the body. They would also monitor for any side effects and check your overall health and vision periodically.

Potential risks and benefits

Participating in this study might offer the potential benefit of a new treatment for CLN2-related eye problems, which could possibly improve or reduce vision loss. However, as this is a new treatment, there are also potential risks, including side effects from the gene therapy itself or from the procedures involved. These risks are not fully known yet, and the study is designed to carefully monitor for them. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

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

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Unverified
Germany

Common questions

What is gene therapy?

Gene therapy is a medical approach that aims to treat diseases by correcting faulty genes, or by providing new genes, to help the body work correctly.

What is CLN2 disease?

CLN2 disease is a rare, inherited condition that affects the brain and nervous system, leading to problems with movement, speech, and vision, primarily in children.

How long will the study last?

The main part of the study where safety and initial effects are checked will last for approximately one year (360 days) after the treatment is given.

Will this treatment cure CLN2 disease?

This early-stage study is primarily focused on safety and seeing if the treatment can help with the eye problems linked to CLN2 disease. It's too early to say if it could be a cure.

What does 'first-in-human' mean?

It means this is the very first time this specific treatment has been given to people, so doctors will be extra careful in checking for safety and how the body reacts.