NCT07054515 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
| NCT ID | NCT07054515 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Azafaros A.G. |
| Condition | Niemann-Pick Type C Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 147 participants |
| Start Date | 2025-06-30 |
| Primary Completion | 2027-11-04 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 147 participants in total. It began in 2025-06-30 with a primary completion date of 2027-11-04.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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
Eligibility Criteria
Inclusion Criteria: * Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725). * Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile/ juvenile onset. Detailed inclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543). Exclusion Criteria: * Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC * Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07054515 clinical trial?
This trial is open to participants of all sexes, aged 4 Years or older, studying Niemann-Pick Type C Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07054515 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 147 participants.
Is NCT07054515 currently recruiting?
Yes, NCT07054515 is actively recruiting participants. Contact the research team at info@azafaros.com for enrollment information.
Where is the NCT07054515 trial being conducted?
This trial is being conducted at Oakland, United States, Minneapolis, United States, Rochester, United States, Dallas, United States and 11 additional locations.
Who is sponsoring the NCT07054515 clinical trial?
NCT07054515 is sponsored by Azafaros A.G.. The trial plans to enroll 147 participants.