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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

RecruitingPhase 3Ages 4 Years - anyAllAzafaros B.V.
NCT07054515 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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

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📋 Eligibility rules (as published)
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
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What it tests

  • AZ-3102
  • Placebo
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Where

39 sites
UCSF Children's Hospital and Research Center at Oakland
Oakland, United States
University of Minnesota Medical School
Minneapolis, United States
Mayo Clinic Childrens Center - PIN
Rochester, United States
Children's Medical Center Dallas
Dallas, United States
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, United States
Hospital Universitario Austral
Ciudad Autónoma Buenos Aires, Argentina
Hospital de Niños de La Santisima Trinidad
Córdoba, Argentina
Women's and Children's Hospital
North Adelaide, Australia

…and 31 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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