Pompe disease: open trials in United Kingdom

• NCT00231400

  Pompe Disease Registry Protocol

  Research · Genzyme, a Sanofi Company

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

  Registry updated 2026-10-28

• 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

  Queen Elizabeth Hospital Birmingham, Birmingham · National Hospital for Neurology & Neurosurgery, London · Royal Free London NHS Foundation Trust, London · Royal Victoria Infirmary, Newcastle upon Tyne · Salford Royal Hospital, Salford

  Registry updated 2026-06-23

• NCT00567073

  Pompe Pregnancy Sub-Registry

  Research · Genzyme, a Sanofi Company

  Cardiff

  Registry updated 2026-04-16

• NCT06121011

  A Global Prospective Observational Registry of Patients With Pompe Disease

  Research · Amicus Therapeutics

  Queen Elizabeth Hospital Birmingham, Birmingham · Cambridge University - Addenbrooke's Hospital, Cambridge · University Hospital of Wales, Cardiff · University Hospital of Wales, Cardiff, Cardiff · Great Ormond Street Hospital NHS Foundation Trust, London · National Hospital For Neurology and Neurosurgery, London · Royal Free Hospital NHS Foundation Trust, Manchester · Salford Royal NHS Foundation Trust, Salford

  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

  Great Ormond Street Hospital For Children NHS Foundation Trust, London

  Registry updated 2026-01-22

All Pompe disease trials and the map

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

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