Product
Miglustat
Aliases
AT2221
5 clinical trials
4 indications
Indication
Pompe DiseaseIndication
GM2 GangliosidosisIndication
Supportive CareClinical trial
A Global Prospective Observational Registry of Patients With Pompe DiseaseStatus: Recruiting, Estimated PCD: 2034-12-20
Clinical trial
A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/PlaceboStatus: Completed, Estimated PCD: 2020-12-15
Clinical trial
An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe DiseaseStatus: Recruiting, Estimated PCD: 2026-06-01
Clinical trial
Survey of Miglustat Therapeutic Effects on Neurological and Systemic Symptoms of Infantile Type of Sandhoff and Taysachs DiseasesStatus: Recruiting, Estimated PCD: 2024-12-15
Clinical trial
An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 YearsStatus: Recruiting, Estimated PCD: 2027-04-01