Pompe disease: open trials in Netherlands

9 open trials have a site in Netherlands. Registry data; only the trial team decides who can take part.

NCT00231400
Pompe Disease Registry Protocol
Research · Genzyme, a Sanofi Company
Rotterdam
Registry updated 2026-10-28
2024-520365-31-01
Pompe disease; new Outcomes measures, Treatment Effect and Novel Targeted Investigations for Advancing Long-term follow-up (POTENTIAL).
Trial Treatment · Phase 4 · Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC), Rotterdam
Registry updated 2026-10-01
2025-522146-40-00
A phase 2, randomized, double-blind study of S-606001 as an add-on to enzyme replacement therapy in patients with late-onset Pompe disease.
Trial Treatment · Phase 2 · Shionogi B.V.
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC), Rotterdam
Registry updated 2026-09-18
NCT07354724
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
Trial Treatment · Phase 1 · Denali Therapeutics Inc.
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC), Rotterdam
Registry updated 2026-08-17
NCT07123155
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Trial Treatment · Phase 2 · Shionogi
Erasmus MC, GE Rotterdam
Registry updated 2026-06-23
NCT06121011
A Global Prospective Observational Registry of Patients With Pompe Disease
Research · Amicus Therapeutics
Dept of Pediatrics Erasmus MC - Sophia Children's Hospital, Rotterdam
Registry updated 2026-03-10
NCT04808505
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Trial Treatment · Phase 3 · Amicus Therapeutics
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC), Rotterdam
Registry updated 2026-01-22
2024-518269-92-00
Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease
Trial Treatment · Phase 4 · Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC), Rotterdam
Registry updated 2024-12-16
NCT06575829
Treatment Frequency Reduction in Pompe Disease
Trial Treatment · Phase 4 · Erasmus Medical Center
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC), Rotterdam
Registry updated 2024-08-28

All Pompe disease trials and the map

This page is not medical advice. Talk to your doctor before contacting a trial.

Report an error