Crigler-Najjar Syndrome: GNT0003 Treatment Study

We are evaluating a new treatment called GNT0003 for adults with severe Crigler-Najjar syndrome who need daily phototherapy. The study aims to see if this treatment is safe and effective for those with pre-existing antibodies.

>2 years16-20 visitsEfficacy phase (II)No PlaceboInvestigational MedicinesOralInjectionHepatologyInternal Medicine

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.

Idefirix
Idefirix is a medicine that removes antibodies to help people with kidney failure receive a donated kidney.
Loratadine Eg
Loratadine Eg is a medicine that helps relieve allergy symptoms like sneezing, runny nose, itchy eyes, and hives.
Prednisolone Eg
Prednisolone Eg is a steroid medicine used to reduce swelling and treat allergies, asthma attacks, and certain immune system 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.

Amoxicillin
Amoxicillin is an antibiotic substance used to treat a range of common bacterial infections such as ear, throat, and urinary tract infections.
Amoxicillin Sodium
Amoxicillin Sodium is an antibiotic substance used to treat common bacterial infections.
Betamethasone Sodium Phosphate
Betamethasone sodium phosphate is a corticosteroid substance that reduces inflammation and suppresses immune reactions in allergic, inflammatory, and skin 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.

RAAV8-Hugt1A1

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.

Adelcort®
Aleric Lora
Alerityn

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.

Loading map...
Assistance Publique Hopitaux De Paris
Pediatrics - Reference center for hereditary diseases of hepatic metabolism
Le Plessis-Robinson, France
Sponsor: Genethon
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.