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NCT07054515 · 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 Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
A Phase 3 study of GM1 Gangliosidosis and GM2 Gangliosidosis, sponsored by Azafaros A.G..
- Recruiting
- Registry status
- Phase 3
- Development phase
- 147
- Enrollment target
- 20
- Study locations
NCT07054515 is a Phase 3 study of GM1 Gangliosidosis and GM2 Gangliosidosis that is actively recruiting participants, run by Azafaros A.G.. The registered enrollment target is 147 participants, above the 83-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (77% higher). The trial reports 20 study locations across 15 states.
The verdict
NCT07054515, a Phase 3 study of GM1 Gangliosidosis and GM2 Gangliosidosis, is actively recruiting participants, sponsored by Azafaros A.G..
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 147 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 Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis
Conditions Studied
Interventions
- DRUG Placebo
- DRUG AZ-3102
Study Locations (20)
Other
- Hospital Pequeno Príncipe - Curitiba
- AP-HP - Hôpital Armand Trousseau - Paris
- SphinCS GmbH - Höchheim
Minnesota
- University of Minnesota Medical School - Minneapolis
- Mayo Clinic Childrens Center - PIN - Rochester
Victoria
- Royal Melbourne Hospital - Parkville
- Royal Children's Hospital Melbourne - PIN - Parkville
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
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 147 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 NCT07054515
The ClinicalTrials.gov registry entry for NCT07054515 describes a study currently listed as recruiting, categorized as Phase 3. The registered enrollment target is 147 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 83-participant average among 6 other GM1 Gangliosidosis trials with a reported enrollment target (77% higher). The listed sponsor is Azafaros A.G., which has 3 total studies on file at ClinicalTrials.gov.
The record links to 3 conditions, with GM1 Gangliosidosis appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT07054515 reports 20 study locations spanning 15 distinct geographic areas - top geographies include Other, Minnesota, Victoria.
Frequently Asked Questions
What is clinical trial NCT07054515 about?
NCT07054515 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 Niemann-Pick Type C Disease, 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 Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gan...
What is the current status of trial NCT07054515?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 147 participants. The study started on 2025-06-30. Estimated completion is 2027-11-04.
What conditions does trial NCT07054515 study?
This clinical trial studies the following conditions: GM1 Gangliosidosis, GM2 Gangliosidosis, Niemann-Pick Type C Disease.
What interventions are being tested in trial NCT07054515?
The interventions under investigation include: Placebo (DRUG), AZ-3102 (DRUG).
Who is sponsoring clinical trial NCT07054515?
This trial is sponsored by Azafaros A.G., which has 3 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07054515 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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Related
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