Miglustat - Clinical Trials

Open-label Study to Evaluate the Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease, With or Without Previous Administration of Miglustat

Enrollment Status: Recruiting

Publish Date: March 19, 2026

Intervention Type: Drug

Study Phase: Phase 2

Summary: This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study (Cohort 1). In addition, the study aims to assess safety, clinical, and biochemical impact of transitioning NPC disease patients to Nizubaglustat after prior treatment with s...

A Global Prospective Observational Registry of Patients With Pompe Disease

Enrollment Status: Recruiting

Publish Date: March 10, 2026

Intervention Type: Other, Biological, Drug

Summary: This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate. The objectives of the registry are: * To evaluate the long-term safety of Pompe diseas...

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 Years

Who is this study for: Pediatric patients with infantile-onset Pompe disease aged 0 to < 18 years

Enrollment Status: Recruiting

Publish Date: January 22, 2026

Intervention Type: Drug, Biological

Study Drugs: Cipaglucosidase alfa, Miglustat

Study Phase: Phase 3

Summary: This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.