- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Interventions
- AZ-3102, Placebo
- Drug
- Lead sponsor
- Azafaros A.G.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 147 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 5
- States / cities
- Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced May 22, 2026, 1:43 AM EDT