Studies how enzyme replacement therapy affects muscle glycogen in Pompe disease
Tests enzyme replacement therapy and its effect on muscle glycogen in adults with Pompe disease.
Plain-language title generated from the registry record
Registry title: GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE
Key facts
- Where
- Copenhagen Oe, DenmarkSee the site
- Phase
- Phase 4
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with genetically confirmed late-onset Pompe disease who have never had enzyme replacement therapy.
- Age
- 18 years or older
- Sex
- All sexes can join.
- Disease stage
- Late-onset Pompe disease, confirmed by genetic testing.
- Earlier treatment
- Must not have had enzyme replacement therapy before.
- Biomarker or test result
- Pompe disease confirmed by genetic testing.
Main reasons for not taking part
- Ferrous (iron-containing) objects in or around the body
- Pacemaker or other implanted electronic device
- Claustrophobia (fear of closed spaces)
- Pregnancy
- Taking part in another trial that may affect the results
Read by machine on 9 November 2026.
Where in Denmark
- RigshospitaletCopenhagen Oe, DenmarkTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record
Print a page for my doctorTrial details
- Who runs it
- Rigshospitalet
- Trial code
- EU 2023-508000-37-01
- Record updated
- 22 August 2025
- Original description
The objective of this trial it to characterize how muscle glycogen is affected by enzyme replacement therapy in treatment naïve patients with Pompe Disease.Read full original descriptionShow less
GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE
The objective of this trial it to characterize how muscle glycogen is affected by enzyme replacement therapy in treatment naïve patients with Pompe Disease.
Inclusion: Subjects with genetically verified Late-onset Pompe Disease; Age ≥18; Naïve to enzyme replacement therapy.
Exclusion: Competing conditions at risk of compromising the results of the study, evaluated by the investigator; Ferrous objects in or around the body; Pacemaker or other implanted electronic devices; Claustrophobia; Inability to understand the purpose of the trial or corporate for the conduction of the experiments; Participation in other trials that may interfere with the results; Pregnancy.