Health TrialsbyHealthPanel

Niemann-Pick Type C Disease

2

Review clinical trials related to Niemann-Pick Type C Disease. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
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]

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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: 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]

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