Europas førende søgning efter kliniske forsøg

TTX-381

Clinical trials are investigating TTX-381 in people with eye-related signs of CLN2 disease. These studies are looking at safety, tolerability, and early signs of benefit in a small group of participants. The main goal is to learn how TTX-381 performs in patients with ocular manifestations of Neuronal Ceroid Lipofuscinosis type 2.

Table of contents

Overview of the TTX-381 study

The clinical trial for TTX-381 is studying people with ocular manifestations of Neuronal Ceroid Lipofuscinosis type 2, also called CLN2 disease.[1] The study is designed to evaluate safety and tolerability, which means the researchers want to learn how well the treatment can be used and what unwanted medical problems may happen.[1]

Who the trial is for

This study is for participants who have eye-related signs of CLN2 disease.[1] The trial includes 22 participants, so it is a small study that is meant to collect early information rather than answer every question about treatment benefit.[1]

Trial phase and study design

The study is a Phase 1/2 trial and is listed as an interventional study.[1] A Phase 1/2 trial is an early clinical study that usually looks first at safety, then begins to look for early signs of how a treatment performs.[1] In this trial, TTX-381 is given by subretinal use, which means it is placed under the retina, the light-sensitive layer at the back of the eye.[1]

What the researchers measure

The main outcome is ocular and overall adverse events and serious adverse events through Day 360.[1] An adverse event is any unwanted medical problem that happens during the study, while a serious adverse event is a more severe problem that may need hospital care or cause major harm.[1] By following participants through Day 360, the researchers are looking beyond the first treatment period to see what happens over time.[1]

Study status and size

The trial is currently Authorised, which means it has been approved to move forward.[1] The planned enrollment is 22 participants, showing that this is a focused study in a rare disease group.[1]

Simple explanations of key terms

Ocular manifestations means eye-related signs or problems caused by a disease.[1] Tolerability means how well people can handle a treatment, including whether side effects are manageable.[1] Interventional means the researchers give a treatment to study its effects, instead of only observing what happens naturally.[1]

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

FAQ

  • What are the TTX-381 clinical trials studying? The trial is studying whether TTX-381 is safe and well tolerated in people with ocular manifestations of CLN2 disease. It also tracks eye-related and overall serious and non-serious unwanted events.
  • Who may take part in the trial? The study is for participants with ocular manifestations of Neuronal Ceroid Lipofuscinosis type 2, also called CLN2 disease. The trial includes a small group of 22 participants.
  • What phase is the TTX-381 study? This is a Phase 1/2 trial. That means it is an early study that looks first at safety and tolerability, while also gathering early clinical information.
  • What condition is being targeted? The trial targets ocular manifestations of CLN2 disease. This means eye-related problems caused by a rare inherited condition.
  • What is the main outcome being measured? The main outcome is eye-related and overall adverse events and serious adverse events through Day 360. An adverse event is any unwanted medical problem that happens during the study.

Igangværende kliniske forsøg for TTX-381

  • Undersøgelse af genterapi med TTX-381 til behandling af øjensymptomer hos patienter med neuronal ceroid lipofuscinose type 2 (CLN2)

    Rekrutterer endnu ikke

    1 1
    Undersøgte lægemidler:
    Tyskland

Ordliste

  • Ocular manifestations: Eye-related signs or problems caused by a disease.
  • CLN2 disease: A rare inherited disease also called Neuronal Ceroid Lipofuscinosis type 2.
  • Neuronal Ceroid Lipofuscinosis: A group of rare inherited disorders that affect the nervous system and can also affect the eyes.
  • Phase 1/2: An early stage of clinical research that first checks safety and tolerability and may also look for early signs of benefit.
  • Interventional study: A study where participants receive a treatment so researchers can learn about its effects.
  • Safety: How well a treatment can be used without causing harmful problems.
  • Tolerability: How well people can handle a treatment, including how manageable side effects are.
  • Adverse event: Any unwanted medical problem that happens during a study, whether or not it is caused by the treatment.
  • Serious adverse event: A more severe unwanted medical problem, such as one that leads to hospital care or causes major harm.
  • Day 360: The study time point 360 days after treatment or study start, used to measure longer-term results.

Referencer

  1. https://kliniske-forsoeg.dk/forsog/afprovning-af-ny-genterapi-ttx-381-til-behandling-af-ojenproblemer-hos-personer-med-cln2-sygdom/