Taking people in Dánia4. fázis
GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE
Key facts
- Where
- Copenhagen Oe, DániaSee the site
- Phase
- 4. fázis
Where in Dánia
- RigshospitaletCopenhagen Oe, DániaTaking 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?
Trial details
- Who runs it
- Rigshospitalet
- Trial code
- EU 2023-508000-37-01
- Record updated
- 2025. augusztus 22.
- 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
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.