Pompe disease: open trials in France

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

NCT00231400
Pompe Disease Registry Protocol
Research · Genzyme, a Sanofi Company
Bordeaux · Brest · Bron · Bron · Caen · La Tronche · Lille · Marseille · Nancy · Nantes · Nice · Nîmes · Paris · Paris · Saint-Priest-en-Jarez · Strasbourg · Toulouse · Tours · Vannes
Registry updated 2026-10-28
NCT06833489
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Trial Treatment · Assistance Publique Hopitaux De Marseille
Hôpital Timone, Marseille
Registry updated 2026-10-28
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.
Hospices Civils de Lyon, Bron · Raymond-Poincare Hospital, Garches · Centre Hospitalier Regional De Marseille, Marseille · Centre Hospitalier Universitaire de Nice, Nice
Registry updated 2026-09-18
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
HLC Hopital Pierre Wertheimer, Bron · AP-HP - hôpital Raymond Poincaré, Garches · Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone, Marseille · CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires, Nice
Registry updated 2026-06-23
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
Raymond-Poincare Hospital, Garches
Registry updated 2026-01-22
NCT03564561
Natural History of Pompe Disease
Research · Assistance Publique - Hôpitaux de Paris
Hôpital Raymond-Poincaré, Garches
Registry updated 2024-04-08

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Pompe disease: open trials in France

Pompe Disease Registry Protocol

Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases

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.

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Natural History of Pompe Disease