Pompe disease trials in Netherlands

In Netherlands 9 Also abroad 30

9 trials · From ClinicalTrials.gov and EU CTIS (euclinicaltrials.eu), checked 11 November 2026. Ask the study team whether a trial fits you.

No phaseTaking people
Pompe Disease Registry Protocol
Where
Rotterdam
People
2,000 worldwide
Run by
Genzyme, a Sanofi Company
See details and how to joinNCT00231400
Phase 4Not yet taking people in Netherlands
Pompe disease; new Outcomes measures, Treatment Effect and Novel Targeted Investigations for Advancing Long-term follow-up (POTENTIAL).
Where
Rotterdam
People
180
Run by
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
See details and how to join2024-520365-31-01 · EU 2024-520365-31-01
Phase 2Taking people
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.
Where
Rotterdam
Age
18 years to 64 years
People
21 worldwide
Run by
Shionogi B.V.
See details and how to join2025-522146-40-00 · EU 2025-522146-40-00
Phase 1Taking people
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
Where
Rotterdam
Age
18 years to 75 years
People
32 worldwide
Run by
Denali Therapeutics Inc.
See details and how to joinNCT07354724 · EU 2025-524082-25-00
Phase 2Taking people
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Where
GE Rotterdam
Age
18 years and over
People
45 worldwide
Run by
Shionogi
See details and how to joinNCT07123155
No phaseTaking people
A Global Prospective Observational Registry of Patients With Pompe Disease
Where
Rotterdam
People
500 worldwide
Run by
Amicus Therapeutics
See details and how to joinNCT06121011
Phase 3Taking people
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Where
Rotterdam
Age
Up to 17 years
People
36 worldwide
Run by
Amicus Therapeutics
See details and how to joinNCT04808505 · EU 2022-501095-25-01
Phase 4Not yet taking people in Netherlands
Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease
Where
Rotterdam
People
156
Run by
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
See details and how to join2024-518269-92-00 · EU 2024-518269-92-00
Phase 4Not yet taking people in Netherlands
Treatment Frequency Reduction in Pompe Disease
Where
Rotterdam
Age
50 years and over
People
10
Run by
Erasmus Medical Center
See details and how to joinNCT06575829 · EU 2024-514255-15-01