Recruiting
Phase 3

Nizubaglustat

Sponsor:

Azafaros B.V.

Code:

NCT07054515

Conditions

Niemann-Pick Type C Disease

GM1 Gangliosidosis

GM2 Gangliosidosis

Eligibility Criteria

Sex: All

Age: 4+

Healthy Volunteers: Not accepted

Interventions

AZ-3102

Placebo

Study Details

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-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis

Conditions

Niemann-Pick Type C Disease

GM1 Gangliosidosis

GM2 Gangliosidosis

Study ID

NCT07054515

Start date

Jun 30, 2025

Status verified date

Jun, 2026

Completion date

Jun 14, 2028

Anticipated

Primary completion date

May 14, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 4+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

147 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Subprotocol NCT07082725: Intervention Group NPC

Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo).

(Recruiting)

experimental: Subprotocol NCT07082543: Intervention Group GM1/GM2

Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo).

(Active, not recruiting - No longer looking for participants)

Interventions

AZ-3102

Oral dispersible tablets

Placebo

A matching placebo will be administered in the same regimen as the intervention

Primary outcome measure

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

Central Contacts and Locations

Central contacts

Patient Advocacy Representative

Please reach out by emailinfo@azafaros.com

Contact for Healthcare Professionals

Please reach out by emailmedinfo@azafaros.com

Locations

UCSF Children's Hospital and Research Center at Oakland

Recruiting

Oakland, California, United States, 94609

Contacts

Veronica Cheung, CRC

510-428-3885Veronica.Cheung@ucsf.edu

Principal Investigator:

Caroline Hastings, MD

Mayo Clinic Childrens Center - PIN

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Brad Jorde

507-293-3609RSTCGRESEARCH@mayo.edu

Children's Medical Center Dallas

Recruiting

Dallas, Texas, United States, 75235

Contacts

Charina Ramirez, MD

214-456-8000charina.ramirez@utsouthwestern.edu

Lysosomal Rare Disorders Research and Treatment Center

Recruiting

Fairfax, Virginia, United States, 22030-7404

Contacts

Lauren Noll

571-732-4655lnoll@ldrtc.org

M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary

Recruiting

Calgary, Alberta, Canada, T3B 6A8

Contacts

Olugbenga Olunaike

+1 587 885 3158olugbenga.olunaike@magiccalgany.ca

More Information

Sponsor

Azafaros B.V.

Last update posted

Aug 14, 2026

Last verified

Jun, 2026

Keywords

  • Nizubaglustat

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Azafaros B.V. on 2026-08-14.