Clinical trials

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23 trials

NCT07354724
Recruiting
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
Phase 1 · Germany, Netherlands, United States · 6 sites
Registry update: 17 Aug 2026
NCT07282847
Recruiting
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)
Phase 1/2 · United States · 9 sites
Registry update: 13 Oct 2026
NCT07664930
Recruiting
Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease
Not applicable · Italy · 1 site
Registry update: 24 Jun 2026
2025-522146-40-00
Recruiting
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.
Phase 2 · Belgium, Germany, Denmark, Spain, France, Italy, Netherlands · 14 sites
Registry update: 18 Sept 2026
NCT07123155
Recruiting
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Phase 2 · Belgium, Germany, Denmark, Spain, France, United Kingdom, Italy, Netherlands, United States · 28 sites
Registry update: 23 Jun 2026
NCT06666413
Recruiting
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Phase 4 · China · 1 site
Registry update: 23 Mar 2026
NCT06833489
Recruiting
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Not applicable · France · 1 site
Registry update: 28 Oct 2026
NCT06391736
Recruiting
Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
Phase 1/2 · China · 1 site
Registry update: 3 Jul 2025
NCT07136844
Recruiting
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
Not applicable · Belgium · 1 site
Registry update: 22 Aug 2025
NCT06121011
Recruiting
A Global Prospective Observational Registry of Patients With Pompe Disease
Not applicable · Austria, Belgium, Germany, Denmark, United Kingdom, Greece, Hungary, Italy, Netherlands, Poland, Slovenia, United States · 41 sites
Registry update: 10 Mar 2026
2023-508000-37-01
Recruiting
GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE
Phase 4 · Denmark · 1 site
Registry update: 22 Aug 2025
NCT05989620
Recruiting
Long-Term Development of Muscular Dystrophy Outcome Assessments
Not applicable · United States · 1 site
Registry update: 18 Nov 2025
NCT07834320
Recruiting
Pompe Disease RWE Long-term Monitoring Program
Not applicable · Taiwan · 1 site
Registry update: 22 Sept 2026
NCT04808505
Recruiting
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Phase 3 · Germany, Denmark, France, United Kingdom, Italy, Netherlands, Taiwan, United States · 18 sites
Registry update: 22 Jan 2026
NCT05734521
Recruiting
Avalglucosidase Alfa Pregnancy Study
Not applicable · United States · 1 site
Registry update: 16 Jan 2026
NCT05619900
Recruiting
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Not applicable · United States · 1 site
Registry update: 8 Apr 2026
NCT04532047
Recruiting
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Phase 1 · United States · 1 site
Registry update: 17 Mar 2026
NCT03564561
Recruiting
Natural History of Pompe Disease
Not applicable · France · 1 site
Registry update: 8 Apr 2024
NCT04639336
Recruiting
Cognitive and Neurological Pathologies in Pompe Disease
Not applicable · United States · 1 site
Registry update: 4 Mar 2026
NCT01665326
Recruiting
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Not applicable · United States · 1 site
Registry update: 8 Apr 2026

Ordered by registry start date. Freshness and sponsorship never change the order.