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Showing 1–3 of 3 matching trials from the live ClinicalTrials.gov search.
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Filters: Intervention: AZ-3102 Clear all
Recruiting Phase 3 Interventional

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

NCT07054515
View directory record Official record
Conditions
Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Sep 2, 2026, 1:04 PM EDT
Recruiting Phase 3 Interventional

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)

NCT07082725
View directory record Official record
Conditions
Niemann-Pick Type C Disease
Interventions
Nizubaglustat, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 16, 2026 · Synced Sep 2, 2026, 1:04 PM EDT
Active, not recruiting Phase 3 Interventional

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

NCT07082543
View directory record Official record
Conditions
Gangliosidoses, GM2, Gangliosidosis, GM1
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Sep 2, 2026, 1:04 PM EDT