Pompe disease trials in China

4 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

  Beijing, Guangzhou, Jinan, Shanghai

  People

  2,000 worldwide

  Run by

  Genzyme, a Sanofi Company

  See details and how to joinNCT00231400
• Phase 4Taking people
  China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

  Where

  Shanghai

  Age

  Up to 17 years

  People

  13

  Run by

  Genzyme, a Sanofi Company

  See details and how to joinNCT06666413
• Phase 1/2Taking people
  Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

  Where

  Beijing

  Age

  6 years and over

  People

  33

  Run by

  GeneCradle Inc

  See details and how to joinNCT06391736
• Early phase 1Not yet taking people in China
  Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)

  Where

  Shanghai

  Age

  18 years and over

  People

  6

  Run by

  Huashan Hospital

  See details and how to joinNCT06178432