MECP2 Duplication Syndrome: ION440 Treatment Study

We are testing a new treatment called ION440 to see if it is safe and well-tolerated in patients with MECP2 Duplication Syndrome. This study will also look at how the drug moves through the body.

Safety phase (I)Efficacy phase (II)Post-Trial Drug AccessNo PlaceboInvestigational MedicinesInjectionHematology

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.

Ionil

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.

Coal Tar
Coal tar is a substance applied to the skin to treat psoriasis, eczema, and dandruff by reducing itching, scaling, and inflammation.
Coal Tar Solution
Coal tar solution is a topical used to treat skin conditions like psoriasis and dandruff by slowing skin cell growth and reducing scaling and itching.
Salicylic Acid
Salicylic acid is a substance that helps remove dead skin and treat acne, warts, and other 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.

Ion440

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.

Acido Salicilico Marco Viti
Acido Salicilico Na
Aco Acnegel

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...
Kepler Universitaetsklinikum GmbH
Department for Pediatrics and Adolescent Medicine
Linz, Austria
Centre Hospitalier Universitaire De Dijon
Centre de Génétique
Saint-Apollinaire, France
Sant Joan De Deu Barcelona Hospital
Neurologia
Esplugues de Llobregat, Spain
Sponsor: Ionis Pharmaceuticals Inc.
Last updated: Nov 26, 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.