Status:
RECRUITING
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
Lead Sponsor:
Azafaros A.G.
Conditions:
Niemann-Pick Type C Disease
GM1 Gangliosidosis
Eligibility:
All Genders
4+ years
Phase:
PHASE3
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...
Detailed Description
This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple disease areas using a Master Protocol Rese...
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
Key Trial Info
Start Date :
June 30 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
November 4 2027
Estimated Enrollment :
147 Patients enrolled
Trial Details
Trial ID
NCT07054515
Start Date
June 30 2025
End Date
November 4 2027
Last Update
April 15 2026
Active Locations (37)
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1
UCSF Children's Hospital and Research Center at Oakland
Oakland, California, United States, 94609
2
University of Minnesota Medical School
Minneapolis, Minnesota, United States, 55455
3
Mayo Clinic Childrens Center - PIN
Rochester, Minnesota, United States, 55905
4
Children's Medical Center Dallas
Dallas, Texas, United States, 75235