Pompe disease trials in United Kingdom

5 trials · From ClinicalTrials.gov, checked 12 November 2026. Ask the study team whether a trial fits you.

• No phaseTaking people
  Pompe Disease Registry Protocol

  Where

  Birmingham, Cambridge, Cardiff, Glasgow, London, Manchester, Newcastle upon Tyne, Salford

  People

  2,000 worldwide

  Run by

  Genzyme, a Sanofi Company

  See details and how to joinNCT00231400
• 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

  Birmingham, London, Newcastle upon Tyne, Salford

  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

  Cardiff

  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

  Birmingham, Cambridge, Cardiff, London, Manchester, Salford

  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

  London

  Age

  Up to 17 years

  People

  36 worldwide

  Run by

  Amicus Therapeutics

  See details and how to joinNCT04808505 · EU 2022-501095-25-01