Taking people in DenmarkPhase 4
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.
Key facts
- Who runs it
- Rigshospitalet
- Where
- Copenhagen Oe, DenmarkSee the site
- Trial code
- Registry title
- GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE
- Status
- Recruiting
- Phase
- Phase 4
- People taking part
- 10 people, planned
- Started
- 13 Feb 2024
- Expected to end
- 31 Dec 2028
- Type of study
- Interventional
- Record updated
- 22 August 2025
Who can take part
AI summary
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
The registry criteria, word for word
Show all 10 criteriaShow less
Who can take part
- Subjects with genetically verified Late-onset Pompe Disease
- Age ≥18
- Naïve to enzyme replacement therapy
Who cannot take part
- 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
Where in Denmark
- RigshospitaletCopenhagen Oe, DenmarkTaking people
About the trial
What the trial wants to find out
The objective of this trial it to characterize how muscle glycogen is affected by enzyme replacement therapy in treatment naïve patients with Pompe Disease.
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 doctorData and sources
- Registry record
- Listed on the EU's official list of trials (CTIS).
- Official title
- GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE
- Plain-language title
- Studies how enzyme replacement therapy affects muscle glycogen in Pompe diseaseGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 9 November 2026.
- Record updated
- 22 August 2025