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NCT07082543 · ClinicalTrials.gov registry record · Phase 3
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
A Phase 3 study of Gangliosidoses, GM2 and Gangliosidosis, GM1, sponsored by Azafaros A.G..
- Recruiting
- Registry status
- Phase 3
- Development phase
- 75
- Enrollment target
- 20
- Study locations
NCT07082543: Recruiting Phase 3 study of Gangliosidoses, GM2 and Gangliosidosis, GM1, sponsored by Azafaros A.G..
NCT07082543 is a Phase 3 study of Gangliosidoses, GM2 and Gangliosidosis, GM1 that is actively recruiting participants, run by Azafaros A.G.. The registered enrollment target is 75 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (90% lower). The trial reports 20 study locations across 16 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07082543, a Phase 3 study of Gangliosidoses, GM2 and Gangliosidosis, GM1, is actively recruiting participants, sponsored by Azafaros A.G..
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 75 participants
- Enrollment target
- 20
- Study locations
Study Summary
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
Primary Outcome
Total SARA comprises eight categories with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia)
Conditions Studied
Interventions
- DRUG Placebo
- DRUG AZ-3102
Study Locations (20)
Other
- Hospital Pequeno Principe - Curitiba
- AP-HP - Hôpital Armand Trousseau - Paris
- SphinCS GmbH - Höchheim
Minnesota
- University of Minnesota Medical School - Minneapolis
- Mayo Clinic Children's Center - PIN - Rochester
Alberta
- M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary - Calgary
- University of Alberta Medical Genetics Clinic - Edmonton
California
- UCSF Children's Hospital and Research Center at Oakland - Oakland
Texas
- Children's Medical Center Dallas - Dallas
Virginia
- Lysosomal Rare Disorders Research and Treatment Center - Fairfax
Buenos Aires
- Hospital Universitario Austral - Ciudad Autónoma Buenos Aires
Córdoba Province
- Hospital de Niños de La Santisima Trinidad - Córdoba
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 75 participants |
| Start Date | 2025-06-30 |
| Est. Completion | 2027-11-04 |
| Phase | Phase 3 |
What NCT07082543 shows while recruiting
NCT07082543 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 75 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (90% lower).
The record links to 2 conditions, with Gangliosidoses, GM2 appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT07082543 names 20 study sites across 16 states, led by Other, Minnesota, Alberta.
Frequently Asked Questions
What is clinical trial NCT07082543 about?
NCT07082543 is a clinical study titled "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". 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
What is the current status of trial NCT07082543?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 75 participants. The study started on 2025-06-30. Estimated completion is 2027-11-04.
What conditions does trial NCT07082543 study?
This clinical trial studies the following conditions: Gangliosidoses, GM2, Gangliosidosis, GM1.
What interventions are being tested in trial NCT07082543?
The interventions under investigation include: Placebo (DRUG), AZ-3102 (DRUG).
Who is sponsoring clinical trial NCT07082543?
This trial is sponsored by Azafaros A.G., which has 3 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07082543 being conducted?
This trial has 20 study locations across California, Minnesota, Texas, Virginia, Buenos Aires. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT07082543's enrollment target sits among peer trials
75 1774th of 2000 higher than 221 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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