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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
NCT07054515 on ClinicalTrials.gov
Study listings come from ClinicalTrials.gov, where sponsors register and update their own studies. A listing does not mean a study is safe or effective, and DatenUS does not recommend any study. Talk with your doctor before joining a clinical trial, and use ClinicalTrials.gov for contact details and full eligibility rules.
- Status
- Recruiting
- Phase
- Phase 3
- Interventional
- Planned enrollment
- 147
- Participants, all sites
- U.S. sites
- 5
- In 4 states
Study details
- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Sponsor
- Azafaros B.V. (Industry)
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Start
- 06/30/2025
- Primary completion (expected)
- 05/14/2028
- Last updated by sponsor
- 09/24/2026
Full eligibility rules, contacts and site names: NCT07054515 on ClinicalTrials.gov
Where the study has sites
Cities with at least one listed U.S. site. Some sites may not be enrolling yet.
- California: Oakland
- Minnesota: Minneapolis, Rochester
- Texas: Dallas
- Virginia: Fairfax
Frequently asked questions
- What does recruiting mean?
- Recruiting means the study is currently enrolling participants. Not yet recruiting means it has been registered but has not started enrolling. Enrolling by invitation means participants are selected by the study team rather than through open enrollment.
- How do I join a clinical trial?
- Read the full eligibility criteria and contact information on ClinicalTrials.gov, then talk with your doctor about whether a study may be right for you. The study team decides who can enroll.
Source: ClinicalTrials.gov, National Library of Medicine · Last updated 09/24/2026