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TTX-381

Clinical trials are investigating TTX-381 in people with ocular manifestations of CLN2 disease. These studies are designed to assess safety, tolerability, and early signs of benefit in a small group of participants. The current trial focuses on subretinal use in a Phase 1/2 study.

Table of Contents

Trial overview

The available study of TTX-381 is an interventional clinical trial in participants with ocular manifestations of Neuronal Ceroid Lipofuscinosis type 2, also called CLN2 disease.[1] The trial is authorised and plans to enroll 22 participants.[1]

This study is focused on the eye-related signs of the disease and uses subretinal use of TTX-381.[1] Subretinal use means the treatment is given under the retina, which is the light-sensitive layer at the back of the eye.

Who can participate

The trial is designed for people who have ocular manifestations of CLN2 disease.[1] In simple terms, this means the study is for patients whose disease affects the eyes.

No other participant groups are listed in the source data, so the trial population is narrowly defined.[1]

Study design and phase

This is a Phase 1/2 study, which is an early stage of clinical research.[1] Phase 1 studies mainly look at safety, while Phase 2 studies also begin to look for early signs that a treatment may help.

The trial brief summary says it aims to evaluate the safety and tolerability of TTX-381 through Day 360 in participants with CLN2 disease.[1]

What the study measures

The primary outcome is ocular and overall adverse events and serious adverse events through Day 360.[1] Adverse events are unwanted medical problems that happen during the study, and serious adverse events are more severe problems that may need urgent care or hospital treatment.

Because this is an early trial, the main focus is on safety and tolerability rather than proving long-term benefit.[1] The follow-up through Day 360 shows that the study is watching participants over a long period after treatment.[1]

Why this trial matters

CLN2 disease is a rare condition, and this study looks at a targeted group of patients with eye involvement.[1] For patients and families, early trials like this are important because they help researchers learn whether a new approach can be studied safely before larger trials are done.

At this stage, the trial data do not report results, so the main information available is about the study plan, the patient group, and the safety outcomes being tracked.[1]

Trial ID Phase Condition studied Status Enrollment
NCT05791864 Phase 1/2 Ocular manifestations of CLN2 disease Authorised 22

FAQ

  • What is being studied in the TTX-381 trial? The trial is studying TTX-381 in people with ocular manifestations of CLN2 disease. It is mainly looking at safety and tolerability over time.
  • Who can take part in the study? The study is for participants with ocular manifestations of Neuronal Ceroid Lipofuscinosis type 2, also called CLN2 disease. The trial is limited to this specific patient group.
  • What phase is the TTX-381 trial? This is a Phase 1/2 study. That means it is an early trial that looks at both safety and early signs of how well the treatment may work.
  • What is the main goal of the study? The main goal is to evaluate ocular and overall adverse events and serious adverse events through Day 360. In simple terms, the study tracks unwanted medical events in the eye and in the whole body.
  • How many people are planned for the trial? The trial plans to enroll 22 participants. This is a small study, which is common in early-phase clinical research.

Ongoing Clinical Trials on TTX-381

  • First study of TTX-381 gene therapy safety in patients with eye problems caused by CLN2 disease (Neuronal Ceroid Lipofuscinosis Type 2)

    Not yet recruiting

    1 1
    Investigated drugs:
    Germany

Glossary

  • Clinical trial: A research study in people that tests a medical treatment to learn more about safety, tolerability, and possible benefit.
  • CLN2 disease: A rare condition called Neuronal Ceroid Lipofuscinosis type 2. It is the disease being studied in this trial.
  • Ocular manifestations: Eye-related signs or problems caused by a disease.
  • Phase 1/2: An early stage of clinical research. Phase 1 focuses mainly on safety, and Phase 2 also begins to look at whether the treatment may help.
  • Safety: Whether the treatment causes harm or unwanted medical problems.
  • Tolerability: How well people can take or receive a treatment without major problems.
  • Adverse event (AE): Any unwanted medical problem that happens during a study, whether or not it is caused by the treatment.
  • Serious adverse event (SAE): A more severe unwanted medical problem, such as one that is life-threatening or needs hospital care.
  • Subretinal use: A way of giving treatment into the area under the retina, which is the light-sensitive layer at the back of the eye.
  • Day 360: The study time point 360 days after treatment or study start, used to measure outcomes over a long follow-up period.

References