Pompe Disease: Enzyme Replacement Therapy Study

We are examining how a new enzyme replacement therapy affects muscle glycogen in people with Pompe disease who have not yet received treatment. This research may help improve future therapies for this condition.

>2 years 6-10 visits Monitoring phase (IV)No Placebo Cost Reimbursement Standard Medicines InjectionEndocrinologyHematologyNeurology

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.

Nexviadyme

Nexviadyme is a medicine used to treat Pompe disease, a rare genetic condition that causes muscle weakness and breathing problems.

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.

Avalglucosidase Alfa

Avalglucosidase alfa is an enzyme replacement substance used to treat Pompe disease by reducing glycogen buildup in muscles to improve breathing and movement.

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.

Myozyme

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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Rigshospitalet

Copenhagen Neuromuscular Center

Copenhagen, Denmark

Sponsor: Rigshospitalet

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.