Status: Recruiting

A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

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 stable, full-dose Miglustat (Cohort 2).

Participants needed: 21

Trial details

Phase: Phase 2Age: 12+Biological sex: AllType: InterventionalSponsor: Azafaros B.V.Updated: Jun 18, 2026Locations: 3

Eligibility criteria

Have been randomized into Phase 2 Study AZA-001-5A2-01. [+5]

A positive serum pregnancy test (only tested for women of childbearing potential... [+3]

Status: Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease

Participants needed: 72

Trial details

Phase: Phase 3Age: 4+Biological sex: AllType: InterventionalSponsor: Azafaros B.V.Updated: Jun 18, 2026Locations: 35

Eligibility criteria

Signed informed consent [+7]

A history of medical conditions other than NPC disease that, in the opinion of t... [+9]

Status: Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis

Participants needed: 147

Trial details

Phase: Phase 3Age: 4+Biological sex: AllType: InterventionalSponsor: Azafaros B.V.Updated: Jun 18, 2026Locations: 38

Eligibility criteria

Male and female participants, aged 4 years and older with a diagnosis of the lat... [+1]

Detailed exclusion criteria are presented in the NPC disease-specific subprotoco... [+1]

Status: Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of GM1 gangliosidosis or GM2 gangliosidosis

Participants needed: 75

Trial details

Phase: Phase 3Age: 4+Biological sex: AllType: InterventionalSponsor: Azafaros B.V.Updated: Jun 18, 2026Locations: 36

Eligibility criteria

Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant [+5]

A history of medical conditions other than GM1 or GM2 gangliosidosis that, in th... [+8]

Clinical trials

A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis