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: Sep 28, 2026Locations: 34

Eligibility criteria

Signed informed consent [+7]

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

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: Sep 24, 2026Locations: 39

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]

Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)

A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).

Participants needed: 20

Trial details

Phase: Phase 2Age: 4-30Biological sex: AllType: InterventionalSponsor: Azafaros B.V.Updated: Jul 20, 2026Locations: 7

Eligibility criteria

Have a confirmed diagnosis of GD3 disease [+5]

Any condition at Screening or Baseline that, in the opinion of the Principal Inv... [+12]

Clinical trials

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

Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)