Natural History of Pompe Disease

Key facts

Sponsor

Assistance Publique - Hôpitaux de Paris

Principal Investigator

Helge Amthor, MD, PhD

Hôpital Raymond Poincaré

Enrollment

20 enrolled

Eligibility

18-80 years · All sexes

Timeline

2019 – 2033

Study locations (1)

• Hôpital Raymond Poincaré, Garches, Hauts-de-Seine, France

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Other trials for Glycogen storage disease due to acid maltase deficiency

Additional recruiting or active studies for the same condition.

• RECRUITINGNANCT07478172
  Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
  University of Missouri-Columbia
• RECRUITINGPHASE1, PHASE2NCT07282847
  A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)
  AskBio Inc
• RECRUITINGPHASE2NCT07123155
  Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
  Shionogi
• RECRUITINGPHASE4NCT06666413
  China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
  Genzyme, a Sanofi Company
• RECRUITINGNCT06795152
  Rare Glycogen Storage Diseases Natural History Study
  Duke University
• RECRUITINGPHASE1, PHASE2NCT06391736
  Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
  GeneCradle Inc
• RECRUITINGNCT06121011
  A Global Prospective Observational Registry of Patients With Pompe Disease
  Amicus Therapeutics
• ACTIVE NOT RECRUITINGEARLY PHASE1NCT06109948
  Study to Assess the Safety, Tolerability, PK and PD of ABX1100
  Aro Biotherapeutics

See all trials for Glycogen storage disease due to acid maltase deficiency →