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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 on ClinicalTrials.gov ↗
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
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07082543
The ClinicalTrials.gov registry entry for NCT07082543 describes a study currently listed as recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 75 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Azafaros A.G., which has 3 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT07082543 reports 20 study locations spanning 16 distinct geographic areas — top geographies include Other, Minnesota, Alberta. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT07082543?
The interventions under investigation include: Placebo (DRUG), AZ-3102 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT07082543?
This trial is sponsored by Azafaros A.G., which has 3 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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