Pompe disease trials in Germany

In Germany 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
Berlin, Bochum, Bonn, Freiburg im Breisgau, Giessen, Halle, Hamburg, Mainz, Munich, Münster
People
2,000 worldwide
Run by
Genzyme, a Sanofi Company
See details and how to joinNCT00231400
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
Halle (Saale), Hochheim Am Main, Munich
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
Hochheim Am Main
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
Halle, Höchheim, München
Age
18 years and over
People
45 worldwide
Run by
Shionogi
See details and how to joinNCT07123155
No phaseTaking people
Pompe Pregnancy Sub-Registry
Where
Bonn
People
20 worldwide
Run by
Genzyme, a Sanofi Company
See details and how to joinNCT00567073
No phaseTaking people
A Global Prospective Observational Registry of Patients With Pompe Disease
Where
Bochum, Höchheim, Marburg, München, Ulm
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
Giessen, Heidelberg, Hochheim Am Main, Münster
Age
Up to 17 years
People
36 worldwide
Run by
Amicus Therapeutics
See details and how to joinNCT04808505 · EU 2022-501095-25-01
No phaseEnrolling by invitation
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Where
München
Age
18 years to 65 years
People
108
Run by
LMU Klinikum
See details and how to joinNCT06605612
No phaseEnrolling by invitation
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
Where
Bad Feilnbach
Age
65 years to 65 years
People
30
Run by
LMU Klinikum
See details and how to joinNCT07072676