Explore our global network of active clinical trials dedicated to fighting Pompe disease. Scientific precision for human breakthrough.
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Pivotal phase III trial that demonstrated the superiority of avalglucosidase alfa (Nexviazyme®) over alglucosidase alfa in respiratory function in patients with LOPD. Completed.
Phase III trial evaluating the safety and efficacy of avalglucosidase alfa in pediatric patients with infantile-onset Pompe disease.
Phase II trial evaluating avalglucosidase alfa in pediatric patients (ages 1 to 18) with Pompe disease previously treated with alglucosidase alfa.
Pivotal phase III trial that evaluated the combination of cipaglucosidase alfa with the oral chaperone miglustat (Pombiliti® + Opfolda®) versus alglucosidase alfa in LOPD. Completed.
Open-label long-term extension study of cipaglucosidase alfa + miglustat in patients who completed prior studies (including PROPEL).
Phase I/II trial evaluating AAV8 vector gene therapy (AT845) administered intravenously in adults with late-onset Pompe disease.
Phase I/II trial of liver-directed AAV vector gene therapy in adult patients with LOPD already receiving ERT.
Phase I/II trial evaluating GC301, a next-generation AAV vector gene therapy, in patients with late-onset Pompe disease.
Phase I trial evaluating S-606001, an oral glycogen synthesis inhibitor, as a potential complementary treatment for Pompe disease.
International observational registry collecting long-term data on the natural history of Pompe disease and outcomes of alglucosidase alfa treatment.
International observational registry for patients treated with cipaglucosidase alfa + miglustat (Pombiliti® + Opfolda®). Collects long-term safety and efficacy data.
French observational study investigating biomarkers to improve diagnosis, prognosis and treatment monitoring of Pompe disease.