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RecruitingPhase 3

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

Azafaros B.V. · NCT07054515

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 4+ · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 3
Target enrollment
147
US sites recruiting
5
Study start
June 30, 2025
Record last updated
September 24, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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

Being studied: AZ-3102; Placebo

Conditions: Niemann-Pick Type C Disease; GM1 Gangliosidosis; GM2 Gangliosidosis

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full 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

Recruiting US locations

  • UCSF Children's Hospital and Research Center at Oakland

    Oakland, CA 94609

    Site contact: Veronica Cheung, CRC · 510-428-3885 · Veronica.Cheung@ucsf.edu

  • University of Minnesota Medical School

    Minneapolis, MN 55455

    Site contact: Melissa Fetterly · 612-672-5151 · melissa.fetterley@fairview.org

  • Mayo Clinic Childrens Center - PIN

    Rochester, MN 55905

    Site contact: Brad Jorde · 507-293-3609 · RSTCGRESEARCH@mayo.edu

  • Children's Medical Center Dallas

    Dallas, TX 75235

    Site contact: Charina Ramirez, MD · 214-456-8000 · charina.ramirez@utsouthwestern.edu

  • Lysosomal Rare Disorders Research and Treatment Center

    Fairfax, VA 22030-7404

    Site contact: Lauren Noll · 571-732-4655 · lnoll@ldrtc.org

Study contacts (from the official record)

  • Patient Advocacy Representative · Please reach out by email · info@azafaros.com
  • Contact for Healthcare Professionals · Please reach out by email · medinfo@azafaros.com
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat… (NCT07054515) | Bored Studies