Trials / Conditions / Late-onset Pompe Disease
Late-onset Pompe Disease
7 registered clinical trials studyying Late-onset Pompe Disease — 1 currently recruiting.
| Status | Trial | Sponsor | Phase |
|---|---|---|---|
| Not Yet Recruiting | A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With La NCT07354724 | Denali Therapeutics Inc. | Phase 1 |
| Active Not Recruiting | Study to Assess the Safety, Tolerability, PK and PD of ABX1100 NCT06109948 | Aro Biotherapeutics | EARLY_Phase 1 |
| Completed | High Dose Inspiratory Muscle Training in LOPD NCT05431127 | Duke University | N/A |
| Terminated | STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT NCT03347253 | Amicus Therapeutics | — |
| Unknown | Search for Serum/Plasma Biomarkers in Pompe's Disease NCT03045042 | Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | — |
| Terminated | A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD) NCT02191917 | BioMarin Pharmaceutical | — |
| Terminated | BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) NCT01924845 | BioMarin Pharmaceutical | Phase 3 |