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NCT07082725 · 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 (NPC)
A Phase 3 study of Niemann-Pick Type C Disease, sponsored by Azafaros A.G..
- Recruiting
- Registry status
- Phase 3
- Development phase
- 72
- Enrollment target
- 20
- Study locations
NCT07082725: Recruiting Phase 3 study of Niemann-Pick Type C Disease, sponsored by Azafaros A.G..
NCT07082725 is a Phase 3 study of Niemann-Pick Type C Disease that is actively recruiting participants, run by Azafaros A.G.. The registered enrollment target is 72 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (91% lower). The trial reports 20 study locations across 17 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07082725, a Phase 3 study of Niemann-Pick Type C Disease, is actively recruiting participants, sponsored by Azafaros A.G..
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 72 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
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 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 |
What NCT07082725 shows while recruiting
NCT07082725 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 72 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (91% lower).
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.
NCT07082725 names 20 study sites across 17 states, led by Victoria, Other, Alberta.
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.
What interventions are being tested in trial NCT07082725?
The interventions under investigation include: Placebo (DRUG), Nizubaglustat (DRUG).
Who is sponsoring clinical trial NCT07082725?
This trial is sponsored by Azafaros A.G., which has 3 total clinical trials registered on ClinicalTrials.gov.
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
Similar trials for Niemann-Pick Type C Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT07082725's enrollment target sits among peer trials
72 1781st of 2000 higher than 217 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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