CTNNB1 Syndrome: Gene Therapy Study for Kids

We are testing a new gene therapy given through an injection in the brain for children with CTNNB1 syndrome. The study aims to see if it is safe and if it may help improve their condition.

1-2 years >20 visits Safety phase (I)Efficacy phase (II)No Placebo Cost Reimbursement Partially Remote Investigational MedicinesNeurologyPediatrics

What diseases are being studied?

Health conditions and diseases that the clinical trial is designed to study and treat.

Who can participate in the clinical trial?

These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..

What medicines are being studied?

Medicines that are officially approved for use in the European Union and considered safe by EMA. These medicines are already on the market and may be tested for new conditions or populations.

Urbason

Urbason is a steroid medicine used to reduce inflammation and treat allergies, asthma, and certain autoimmune or inflammatory conditions.

What active substances are being studied?

Active substances that are officially approved for use in the European Union and considered safe by EMA. These substances are already on the market and may be tested for new conditions or populations.

Methylprednisolone

Methylprednisolone is a corticosteroid that reduces inflammation and calms an overactive immune system.

Methylprednisolone Sodium Succinate

Methylprednisolone sodium succinate is a corticosteroid that reduces inflammation and calms the immune system in severe allergic, inflammatory, or autoimmune conditions.

What experimental substances are being tested?

Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.

Adeno-associated viral vector serotype 9

What similar medicines might you know?

Don't worry if you don't know the medicines tested in the trial. There is a chance you have heard about the similar medicines. Check the list below to see if you are familiar with any of them.

Lemod Solu

Lyo-Drol

Medrol

Where is the clinical trial being conducted?

Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.

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University Medical Center Ljubljana

Department of Pediatric Neurology

Ljubljana, Slovenia

Sponsor: Fundacija CTNNB1 Ustanova Za Raziskave Na Podrocju Genske Terapije

Last updated: Nov 27, 2025

Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.