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
Part of paid clinical trials in Oakland, California.
- Sponsor
- Azafaros A.G.
- Study ID
- NCT07082543
- Phase
- PHASE3
- Status
- Recruiting
Conditions
- Gangliosidoses, GM2
- Gangliosidosis, GM1
Eligibility Criteria
- Sex
- ALL
- Age
- 4 Years - N/A
- Healthy Volunteers
- Not accepted
Interventions
- AZ-3102 — DRUGNizubaglustat
- Placebo — DRUGPlacebo
Study Details
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
Key Dates
- Start date
- Jun 30, 2025
- Status verified
- Sep 2025
- Primary completion
- Nov 4, 2027
- Completion
- Nov 4, 2027
Study Design
- Enrollment
- 75 participants (estimated)
- Allocation
- RANDOMIZED
- Intervention model
- PARALLEL
- Primary purpose
- TREATMENT
Arms
- Experimental: NizubaglustatOnce daily oral dispersible tablets
- Placebo Comparator: Placebo
Primary Outcome Measure
Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score [ Time Frame: Baseline to month 18 ]
Central Contacts
- Patient Advocacy RepresentativePlease reach out by email
- Contact for Healthcare ProfessionalsPlease reach out by email
Locations (5)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| UCSF Children's Hospital and Research Center at Oakland | Oakland | California | 94609 | Caroline Hastings, MD (PRINCIPAL_INVESTIGATOR) |
| University of Minnesota Medical School | Minneapolis | Minnesota | 55455 | - |
| Mayo Clinic Children's Center - PIN | Rochester | Minnesota | 55905 | - |
| Children's Medical Center Dallas | Dallas | Texas | 75235 | - |
| Lysosomal Rare Disorders Research and Treatment Center | Fairfax | Virginia | 22030-7404 |
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