Angelman Syndrome: GTX-102 Treatment Study

We are investigating the long-term safety and effectiveness of GTX-102 for individuals with Angelman Syndrome. The study looks at improvements in cognitive function, communication, and motor skills.

Confirmation phase (III)No Placebo Investigational Medicines InjectionNeurology

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 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.

Anhydrous

Calcium Chloride

Calcium chloride is a substance that rapidly raises blood calcium levels to treat low calcium and restore electrolyte balance.

Calcium Chloride Dihydrate

Calcium chloride dihydrate is a substance used to quickly raise low blood calcium levels and help stabilize heart function in emergencies.

What experimental substances are being tested?

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

Elliotts B Solution

Gtx-102

Gtx/ux Diluent and Flush Solution

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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Centre Hospitalier Regional De Marseille

Hôpital d’enfants de la Timone

Marseille, France

Hopital Necker Enfants Malades

Neurologie Pédiatrique

Montrouge, France

University Medical Center Hamburg-Eppendorf

Pediatric Neurology

Hamburg, Germany

Sponsor: Ultragenyx Pharmaceutical 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.