GTX-102 for Children with Angelman Syndrome

We are studying whether GTX-102 can improve cognitive function and other skills in children with deletion-type Angelman Syndrome. The trial also aims to evaluate the safety of this new treatment.

>2 years Confirmation phase (III)Post-Trial Drug Access No Placebo Cost Reimbursement Partially Remote Investigational Medicines InjectionNeurologyPediatrics

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.

Calcium Chloride Dihydrate

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

Disodium Phosphate

Disodium phosphate is a salt used to replace phosphate and restore electrolyte balance.

Magnesium Sulfate Heptahydrate

Magnesium sulfate heptahydrate is a substance used to replace low magnesium and help prevent seizures in pregnant women with severe preeclampsia.

What experimental substances are being tested?

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

Gtx-102

Gtx/ux Diluent And Flush Solution

C-Methylene-P-Thio-Adenylyl

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 Hamburg-Eppendorf

Hamburg, Germany

Universitaet Leipzig

Leipzig, Germany

Ludwig-Maximilians-Universitaet Muenchen

Munich, 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.