Oral nizubaglustat (AZ-3102) for late‑infantile and juvenile Niemann‑Pick C, GM1, and GM2 gangliosidoses

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This randomized, double‑blind, placebo‑controlled Phase 3 Master Protocol tests oral nizubaglustat (AZ‑3102) over 18 months in participants with late‑infantile or juvenile NPC, GM1, or GM2 gangliosidosis. Participants are assigned to disease‑specific subprotocols and randomized to receive active drug or placebo, with detailed procedures and endpoints defined per subprotocol. The study is multicenter and includes planned safety monitoring, efficacy assessments, and disease‑specific outcome measures. Specific eligibility and exclusion criteria differ by subprotocol and are described in the corresponding clinicaltrials.gov records.

Why it matters

Who can join: the full rules

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

Where it's running

• UCSF Children's Hospital and Research Center at Oakland
  Oakland, California, United States Recruiting
• University of Minnesota Medical School
  Minneapolis, Minnesota, United States Recruiting
• Mayo Clinic Childrens Center - PIN
  Rochester, Minnesota, United States Recruiting
• Children's Medical Center Dallas
  Dallas, Texas, United States Recruiting
• Lysosomal Rare Disorders Research and Treatment Center
  Fairfax, Virginia, United States Recruiting
• Hospital de Niños de La Santisima Trinidad
  Córdoba, Córdoba Province, Argentina Recruiting

Study contacts

• Study coordinator: Patient Advocacy Representative
• Email: info@azafaros.com
• Phone: Please reach out by email

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Oral nizubaglustat (AZ-3102) for late‑infantile and juvenile Niemann‑Pick Type C

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• Evaluation of markers in Niemann-Pick Disease, Type C

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.