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

Part of paid clinical trials in Oakland, California.

Sponsor
Azafaros A.G.
Study ID
NCT07054515
Phase
PHASE3
Status
Recruiting
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Conditions

  • GM1 Gangliosidosis
  • GM2 Gangliosidosis
  • Niemann-Pick Type C Disease

Eligibility Criteria

Sex
ALL
Age
4 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • AZ-3102 — DRUG
    Oral dispersible tablets
  • Placebo — DRUG
    A matching placebo will be administered in the same regimen as the intervention

Study Details

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

Key Dates

Start date
Jun 30, 2025
Status verified
Jun 2026
Primary completion
Nov 4, 2027
Completion
Nov 4, 2027

Study Design

Enrollment
147 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Subprotocol NCT07082725: Intervention Group NPC
    Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)
  • Experimental: Subprotocol NCT07082543: Intervention Group GM1/GM2
    Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)

Primary Outcome Measure

Number of participants allocated to subprotocol AZA-001-301-NPC [ Time Frame: Baseline to month 18 ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
UCSF Children's Hospital and Research Center at OaklandOaklandCalifornia94609
Veronica Cheung, CRC
[email protected]
510-428-3885
Caroline Hastings, MD (PRINCIPAL_INVESTIGATOR)
University of Minnesota Medical SchoolMinneapolisMinnesota55455-
Mayo Clinic Childrens Center - PINRochesterMinnesota55905-
Children's Medical Center DallasDallasTexas75235-
Lysosomal Rare Disorders Research and Treatment CenterFairfaxVirginia22030-7404
Lauren Noll
[email protected]
571-732-4655

Find similar trials in Oakland, CA

By research site
UCSF Children's Hospital and Research Center at Oakland· Oakland, CAUniversity of Minnesota Medical School· Minneapolis, MNMayo Clinic Childrens Center - PIN· Rochester, MNChildren's Medical Center Dallas· Dallas, TXLysosomal Rare Disorders Research and Treatment Center· Fairfax, VA

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