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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 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. The trial reports 20 study locations across 16 states.
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
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 |
Interested in This Trial?
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, categorized as Phase 3. 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.
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 reports 20 study locations spanning 16 distinct geographic areas - top geographies include 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.
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