Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 (NPC)
NCT07082725 · 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 Niemann-Pick type C disease
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Nizubaglustat
Study Locations (20)
Victoria
- Royal Melbourne Hospital — Parkville
- Royal Children's Hospital Melbourne - PIN — Parkville
Other
- Hospital Pequeno Principe — Curitiba
- SphinCS GmbH — Höchheim
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
Minnesota
- University of Minnesota Medical School — Minneapolis
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 | 72 participants |
| Start Date | 2025-06-30 |
| Est. Completion | 2027-11-04 |
| Phase | Phase 3 |
Interested in This Trial?
Always speak with your doctor before enrolling in a clinical trial.
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07082725
The ClinicalTrials.gov registry entry for NCT07082725 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 72 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 1 condition, with Niemann-Pick Type C Disease 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: NCT07082725 reports 20 study locations spanning 17 distinct geographic areas — top geographies include Victoria, Other, 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 NCT07082725 about?
NCT07082725 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 (NPC)". 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
What is the current status of trial NCT07082725?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 72 participants. The study started on 2025-06-30. Estimated completion is 2027-11-04.
What conditions does trial NCT07082725 study?
This clinical trial studies the following conditions: Niemann-Pick Type C Disease. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT07082725?
The interventions under investigation include: Placebo (DRUG), Nizubaglustat (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT07082725?
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 NCT07082725 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 Clinical Trials Work
Understand phases 1-4, trial design, randomization, and the informed consent process.
Patient Rights in Clinical Trials
Your rights as a participant: consent, withdrawal, privacy, and who to contact.
Finding the Right Clinical Trial
A practical guide to searching trials, understanding eligibility, and evaluating options.
All Guides
Browse our complete library of clinical trial educational resources.
Read our methodology — how this data is sourced, computed, and verified.